Banks, health care companies lead stocks slightly higher – STLtoday.com

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Updated at 6:17 p.m.

NEW YORK A late wave of buying helped nudge U.S. stock indexes slightly higher Friday after a day of mostly listless trading.

Banks and health care stocks climbed the most as investors priced in an increasing likelihood that interest rates will rise in the coming months.

Federal Reserve Chair Janet Yellen helped stoke those expectations in a speech in which she said an improving job market and rising inflation would likely prompt the central bank to increase borrowing costs.

"The real takeaway here is if the Fed is willing to start moving, they see the economy as not only doing better but likely to do better going forward," said Brad McMillan, chief investment officer at Commonwealth Financial Network. "The Fed is notorious for waiting until the evidence of growth is absolutely undeniable."

The Dow Jones industrial average rose 2.74 points, or 0.01 percent, to 21,005.71. The Standard & Poor's 500 index gained 1.20 points, or 0.1 percent, to 2,383.12. The Nasdaq composite index added 9.53 points, or 0.2 percent, to 5,870.75. Small-company stocks fell. The Russell 2000 index slipped 1.54 points, or 0.1 percent, to 1,394.13.

Speaking in Chicago on the Fed's economic outlook Friday, Yellen said the Fed will likely resume raising interest rates later this month to reflect a strengthening job market and inflation edging toward the central bank's 2 percent target rate.

Yellen added that the central bank expects steady economic improvement to justify additional rate increases. While not specifying how many rate hikes could occur this year, Yellen noted that Fed officials in December had estimated that there would be three this year.

Investors' expectations of a rate hike this month had been building in recent days as remarks by other Fed officials signaled the central bank is ready to resume raising rates as soon as its next two-day meeting of policymakers on March 14-15.

That's one reason the major indexes moved little before and after Yellen's speech.

Still, the increased likelihood of higher interest rates gave several stocks a modest lift, including banks, which stand to make healthier profits from lending as rates rise. Bank of the Ozarks added $1.09, or 2 percent, to $56.24, while Signature Bank rose $2.79, or 1.7 percent, to $162.24.

Not faring as well were real estate, utilities and phone company stocks, which tend to lose favor among yield-seeking investors when interest rates rise.

"If yields are going up you don't need to buy those stocks to get your yield, you just buy 10-Year Treasury notes," said John Canally, chief economic strategist for LPL Financial.

Bond prices were little changed after pulling back from an early climb. The 10-year Treasury yield held steady at 2.48 percent.

Wall Street's slight gains on Friday left the stock market hovering near its latest record highs set on Wednesday.

Stronger-than-expected earnings from companies, continued improvement in the U.S. economy and expectations for business-friendly policies from Washington have helped propel the market this year to new highs. Should investors be nervous about a pullback?

"In the very short term there is some risk of a pullback," said Randy Frederick, vice president of trading and derivatives at the Schwab Center for Financial Research. "I wouldn't say it's likely to approach anything close to a correction, or a 10 percent pullback. Long-term, we continue to think we're solidly in a bull market."

Airlines were among the stocks that notched solid gains Friday.

American Airlines Group rose $1.10, or 2.4 percent, to $46.82, while Alaska Air Group added $2.58, or 2.7 percent, to $98.94. United Continental picked up $2.31, or 3.2 percent, to $75.59.

Disappointing company earnings and outlooks pulled down several stocks.

Costco fell $7.72, or 4.3 percent, to $170.26. Firearms manufacturer American Outdoor Brands, formerly called Smith & Wesson, declined 55 cents, or 2.8 percent, to $18.83.

Revlon slid $1.40, or 4.1 percent, to $32.65, while L Brands, the parent of Victoria's Secret, fell $1.07, or 2 percent, to $52.34.

Macy's also fell sharply, one of several retailers that closed lower Friday. The department store chain declined the most among stocks in the S&P 500, skidding $1.45, or 4.4 percent, to $31.77.

Big Lots bucked the trend, climbing 3.8 percent after the discount retailer reported a larger profit than analysts expected. The stock added $1.98 to $54.23.

Major indexes in Europe were mixed. Germany's DAX fell 0.3 percent, while France's CAC 40 rose 0.6 percent. Britain's FTSE slipped 0.1 percent. Earlier in Asia, Japan's Nikkei 225 index fell 0.5 percent, while South Korea's Kospi sank 1.1 percent. Hong Kong's Hang Seng index lost 0.7 percent.

Energy futures rose. Benchmark U.S. crude gained 72 cents, or 1.4 percent, to close at $53.33 a barrel in New York. Brent crude, used to price international oils, added 82 cents, or 1.5 percent, to close at $55.90 a barrel in London. Wholesale gasoline picked up a penny to close at $1.65 a gallon. Heating oil added a penny to close at $1.59 a gallon. Natural gas rose 2 cents to close at $2.83 per 1,000 cubic feet.

The dollar fell to 114.04 yen from 114.51 yen on Thursday. The euro rose to $1.0599 from $1.0502.

Gold fell $6.40 to $1,226.50 an ounce. Silver slipped a penny to $17.70 an ounce. Copper rose a penny to $2.69 a pound.

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Banks, health care companies lead stocks slightly higher - STLtoday.com

Conservative groups, lawmakers demanding ‘full repeal’ could derail health care rollback – Chicago Tribune

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An array of conservative lawmakers, organizations and activists are demanding a swifter and more aggressive remake of the Affordable Care Act than many Republicans are comfortable with, raising questions about whether President Trump and the GOP are headed toward gridlock as they try to fulfill their promise to repeal the health-care law.

Three conservative senators known for bucking GOP leadership during Barack Obama's presidency - Ted Cruz (Texas), Rand Paul (Ky.) and Mike Lee (Utah) - are raising the possibility of doing the same under Trump.

And outside Congress, three prominent groups - FreedomWorks, Americans for Prosperity and Heritage Action for America - plan to increase pressure on lawmakers to repeal the law fully or risk retribution from the conservative grass roots.

If they hold together in the Senate, where Republicans have just 52 seats, the three senators alone could sink a Republican bill.

The current proposal, floated privately this week by House Republicans, repeals portions of the ACA but, due to pressure from constituents who depend on the law, leaves some elements intact that conservatives are not happy about. Few details of the proposal have emerged publicly.

"The repeal bill ought to be a repeal," Paul said Thursday, as he declared about a replacement plan House Republicans presented to GOP senators at a closed-door meeting the previous afternoon. He also raised the possibility that Cruz and Lee might join him. "Talk to the two people that tweeted out with me," he said.

Cruz and Lee used similar language in tweets this week. With reporters, Cruz has been more circumspect, but he has left open the possibility of opposing the Republican plan. "There's agreement and disagreement between the two chambers, but at the end of the day, I believe we will repeal Obamacare," he said.

In addition to starting a game of chicken with Republican leaders on the Hill and the Trump administration, opponents of anything less than full repeal have also created uncertainty for millions of Americans who receive coverage through the ACA.

The strife came as House Speaker Paul Ryan, R-Wis., laid out a three-week timeline for the passage of health-care legislation in a closed-door meeting with fellow Republicans Thursday, according to numerous attendees.

For the many Republicans who were elected during Obama's presidency with a mandate to block his agenda, obstruction comes much more naturally than governance. The effort to repeal the ACA is the first major test of whether they can harness the energy they used to oppose the law to actually undo it - or whether ideological divisions will sink the effort.

The coordinated resistance has raised the spectre of a resurgent ideological right wing, which has appeared at least publicly to be in retreat since Trump's victory. Many of the president's positions, including his desire to protect insurance coverage for Americans, run counter to conservative orthodoxy and leave room for a revolt.

But Trump's continued popularity on the right puts these conservatives in a tough spot. should 2the president more fully embrace the emerging House plan.They risk alienating Trump's loyal base - a prospect many lawmakers do not take lightly.

"I don't want to draw a line and say that I'm against this proposal and I will put a 'no' vote up," said Rep. Steve King, R-Iowa, who prefers full repeal.

The House plan calls for a refundable tax credit to help Americans afford insurance premiums, but conservatives in the House and the Senate think it amounts to an expensive new federal entitlement.

Key House committees are set to take up legislation as soon as next week. The first steps involve parallel action by the House Ways and Means Committee and the House Energy and Commerce Committee.

The following week, the House Budget Committee is scheduled to combine the bills into a "reconciliation" package eligible for Senate debate, with votes on the House floor expected the week after that.

No legislative text has been released by Ryan's office or by the relevant committees. One part of the legislation, handled by the Energy and Commerce Committee, has been made available to members of that panel - but only for inspection behind closed doors.

Paul complained Thursday that House GOP leaders were being too secretive. Democrats voiced similar complaints.

"We're here today because I'd like to read the Obamacare bill," said Paul near the room where the bill was being reviewed. "If you'd recall, when Obamacare was passed in 2009 and 2010, Nancy Pelosi said, 'You'll know what's in it after you pass it.' The Republican Party shouldn't act in the same way."

Paul, Cruz and Lee are not the only ones who oppose some details of the House plan. Some House conservatives, including King, don't like what they have seen and have embraced alternative ideas.

Conservative Republicans have long opposed refundable tax credits because Americans with lower incomes, who pay less in taxes, receive the full credit even if it exceeds their tax bill. Nonrefundable credits can be used only to offset actual tax liability - but would also mean less money in the pockets of Americans who need help paying for health insurance.

As a result of that dispute and others, conservatives have slowly built support for a "full repeal" plan since the start of the year. Paul provided the only Republican "no" vote on January's non-binding budget reconciliation instructions, saying that it would add too much to the national debt; at the time, Lee and Cruz co-signed a letter saying they would oppose a later bill if it did not repeal the ACA.

Conservatives hailed the apparent unity of Paul, Lee and Cruz on pushing for a full repeal - a model based on legislation that passed Congress in 2015 only to be vetoed then-President Obama.

"If people don't credibly think there are 51 votes for a plan, then the plan doesn't go forward," said Michael Needham, the president of Heritage Action for America, speaking of the Senate. "It's very helpful to have this bloc in the Senate, and in the House, saying they're not going to take less than they got in 2015."

At a Heritage Foundation-sponsored roundtable event with House Freedom Caucus members, Lee said that a repeal bill "should not be anything less aggressive than what we were able to pass in 2015."

To many Republicans, the current conflict triggers the feeling of deja vu. The House Freedom Caucus had issued threats to oppose Republican budgets and to unseat then-House Speaker John Boehner, R-Ohio; Cruz had floated the idea of a government shutdown over Planned Parenthood funding, then backed off.

In interviews, opponents of the current House proposal, which they call "Obamacare-lite," argued that this fight is different. "This has been baking for seven years," said Rep. Mark Sanford, R-S.C., a Freedom Caucus member. Fights in previous years "didn't have the political urgency that repeal does."

Cruz held forth with reporters outside the Senate chamber for 10 minutes earlier this week, seeming to relish in the chance to criticize the House leadership's guidelines and pitch an alternative.

"If we fail to honor our commitment to repeal Obamacare, I believe the consequences would be quite rightly catastrophic," Cruz warned this week in the same apocalyptic tone he often he used as a presidential candidate.

Cheered by that kind of rhetoric, and planning their own push for full repeal, conservative groups have promised to wage a public campaign against Republicans who buckle and save parts of the ACA.

"We're going to be more strongly reminding Republicans of their promises made over the last eight years on the issue of stopping - or at least rolling back, anyway - government-run health care," said Tim Phillips, the president of Americans for Prosperity (AFP). "We're telling them to keep their promises - and they've promised an unequivocal repeal of Obamacare."

Founded by the billionaire donor David Koch, AFP has become an effective grass-roots organization, stopping Republican legislators in Florida, Tennessee and Virginia from expanding Medicaid under the provision of the ACA or building health insurance exchanges. AFP, Phillips said, would demand that lawmakers pass full repeal "both in Washington in a very vocal way" and "also back home in their districts." He declined to be more specific.

Adam Brandon, the president of FreedomWorks, said the group is organizing a "day of action" on March 15, with activists flooding Capitol Hill to "put the heat" on Republicans who don't support full repeal. They take it as a given that the Cruz/Lee/Paul troika will be with them.

"They're damn serious," he said. "It's completely possible that the Ryan-Trump plan, when there is a plan, gets dropped. My jaw kind of hits the floor when I think that we're even having a conversation about this."

On the other hand, some Republicans think they can whittle down the conservative opposition as the chance of repealing the ACA, in part or entirely, becomes more real. Sen. Bill Cassidy, R-La., the sponsor of a bill that would allow states to keep most of the ACA if they want to, told reporters this weekthat Republicans could fulfill their promises if they repealed the most controversial parts of the law.

Cassidy said the mission of Republicans in Congress is not to pitch their ideal plans, but to get right with what the president ran on.

"The American people voted for his vision," he said. "More than any other single person in our country right now, he is in sync with the national mood. If folks want to go their own way, maybe they should run for president."

As the reporters assembled around, Cassidy began to laugh.

"Maybe they did," he said.

The Washington Post's Kelsey Snell contributed to this report.

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Conservative groups, lawmakers demanding 'full repeal' could derail health care rollback - Chicago Tribune

UC Merced professor finds genetic triggers related to cancer – Merced Sun-Star

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Merced Sun-Star
UC Merced professor finds genetic triggers related to cancer
Merced Sun-Star
Everybody has a similar set of genes, but what makes us unique is their activity and, in a way, how the information is expressed, Filipp said. Targeting those newly identified genes and pathways could give researchers a new avenue for precision ...

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UC Merced professor finds genetic triggers related to cancer - Merced Sun-Star

Will Sickle Cell Be the Next Disease Genetic Engineering Cures? – Gizmodo

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Sickle cell disease. Image: Flickr

Sickle cell disease is a slow, vicious killer. Most people diagnosed with the red blood cell disorder in the US live to be between 40 and 60. But those years are a lifetime of pain, as abnormal, crescent-shaped hemoglobin stops up blood flow and deprives tissues of oxygen, causing frequent bouts of agony, along with more severe consequences like organ damage. Now, after decades of searching for a cure, researchers are announcing that, in at least one patient, they seem to have found a very promising treatment.

Two years ago, a French teen with sickle cell disease underwent a gene therapy treatment intended to help his red blood cells from sickling. In a paper published Thursday in the New England Journal of Medicine, the researchers revealed that today, half of his red blood cells have normal-shaped hemoglobin. He has not needed a blood transfusion, which many sickle cell patients receive to reduce complications from the disease, since three months after his treatment. He is also off all medicines.

To reiterate, the paper is a case study of just one patient. Bluebird Bio, the Massachusetts biotech company that sponsored the clinical trial, has treated at least six other trials underway in the US and France, but those results have not yet been fully reported. The gene therapy has not worked quite as well in some of those other patients; researchers say they are adjusting the therapy accordingly. It is also possible that the boy may eventually experience some blood flow blockages again in the future.

The results, though early, are encouraging. They represent the promise of new genetics technologies to address a disease that has long been neglected and tinged with racism. Sickle cell disease affects about 100,000 people in the US, most of whom are black. It is an inherited genetic disease caused by a mutation of a single letter in a persons genetic code.

This single-letter mutation makes it a promising candidate for cutting edge technologies, like the gene-editing technique CRISPR-Cas9, and other gene therapies. Recently, a rush of new research has sought to address it. Two other gene therapy studies for sickle cell are underway in the US one at UCLA and another at Cincinnati Childrens Hospital. Yet another is about to start in a collaboration between Harvard and Boston Childrens Hospital. Last fall, researchers all demonstrated the ability to correct the mutation in human cells using CRISPR, though that strategy will yet have to surpass significant scientific and political hurdles before reaching clinical trials.

In the new study, researchers took bone marrow stem cells from the boy and fed them corrected versions of a gene that codes for beta-globin, a protein that helps produce normal hemoglobin. The hope was that those altered stem cells would interfere with the boys faulty proteins and allow his red blood cells to function normally. They continued the transfusions until the transplanted cells began to produce normal-shaped hemoglobin. In the following months, the numbers of those cells continued to increase until in December 2016, they accounted for more than half the red blood cells in his body. In other words, so far so good.

Currently, the only long-term treatment for sickle cell disease is a bone marrow transplant, a high-risk, difficult procedure which many patients are not even eligible for. Pain and other side-effects are treated with blood transfusions for temporary relief. New technologies offer the hope of a solution that could provide long-term relief and allow patients to live some semblance of a normal life.

For decades, gene therapies have been touted as a cure for everything. But so far, successes have been infrequent, and often for very rare diseases. But early success in treating sickle cell disease means that soon, if were lucky, the benefits of this technology may reach hundreds of thousands of people.

[New England Journal of Medicine]

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Will Sickle Cell Be the Next Disease Genetic Engineering Cures? - Gizmodo

Sickle cell anemia patient ‘cured’ by gene therapy, doctors say – CNN

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"Since therapy was applied, he hasn't had any pain, any complications. He is free of any transfusions. He plays sports and goes to school," said Dr. Philippe Leboulch, an author of the new research and a professor of medicine at the University of Paris. "So we are quite pleased with the results."

This success provides proof of concept for human patients, Leboulch said.

According to Dr. Marina Cavazzana, senior author of the study and head of the biotherapy department at Necker, "all the biological tests we perform lead us to think he is cured." Yet, she added, the answer to the question of whether he is truly cured "can be provided only by the longer follow-up."

Still, hopes are running high that patients with this very devastating disease can receive this therapy "in the next five years," Cavazzana said. "This is our hope, and we work very hard to attain it."

Worldwide, more than 275,000 infants are born with sickle cell disease each year. In the United States, approximately 100,000 people, most of African ancestry or identifying as black, currently have it. About one in every 365 black children in the US is born with sickle cell disease, for which the life expectancy is now about 40 to 60 years.

Sickle cell disease is one of the most common gene disorders in the world, explained Leboulch. A genetic mutation causes hemoglobin, the main constituent of red blood cells, to distort the shape of the cell, and this causes the blood to aggregate or clog.

This leads to "tremendous pain, anemia and also lesions of organs that ultimately result in shortness of life expectancy," Leboulch said. "So what we did here was, we tried to inhibit the process of aggregation."

Essentially, researchers extracted bone marrow from the patient, harvested the stem cells and altered the genetic instructions so that they would make normal hemoglobin. Next, they treated the patient with chemotherapy for four days to eliminate his diseased stem cells. Finally, they returned the treated stem cells via an IV into his bloodstream.

"At that point, the new cells that were modified outside the body started to make new blood cells, and we hope this will be stable for the life of the patient," Leboulch said.

Before receiving treatment, the teen had terrible pain and needed blood transfusions, which required twice-yearly hospitalizations, Leboulch explained. His many complications included necrosis of the hip, which necessitated hip replacement surgery.

Going forward, the plan is to proceed through clinical trials and, if results are promising, make the treatment available to patients. Leboulch and his colleagues are using the same genetic therapy to treat a similar disease called thalassemia, another inherited blood disorder in which patients have less hemoglobin and fewer red blood cells than normal. Severe forms require regular blood transfusions.

Leboulch and his colleagues have global phase 2 and phase 3 trials for the thalassemia treatment underway in France, the US, Australia and Thailand.

For sickle cell disease, a companion trial in the US is underway. "I understand that seven (sickle cell) patients have been treated already. Of course, the outcome is much shorter, and we don't have the results just yet, but it's coming along," Leboulch said.

"To apply this to a baby or a very young child should be at least as effective or more," he said. "Doing it with older patients, who have had years of complications, could be more challenging."

According to Dr. Alexis Thompson, president-elect of the American Society of Hematology, the majority of sickle cell disease patients do not have a sibling who would be an appropriate match for bone marrow donation.

"Gene therapy holds promise because a patient serves as his own donor," and the "risks are much reduced" since there's no possibility of a mismatch, said Thompson, who was not involved in this research but is an investigator on a related gene therapy study.

"I think this is a really very exciting advancement," she said, adding that if the results seen in France can be duplicated, this would provide "for a new direction for patients who need a curative option."

"Gene therapy offers hope for all patients with sickle cell disease, regardless of whether they have a bone marrow match or not," Wong wrote in an email.

"Time is still needed" to assess the success of this treatment and the possibility of later side effects, said Wong. "But any patient with severe sickle cell disease will tell you that being able to live a life for even a year without medications or fear of pain or hospitalization is substantial."

Leboulch also feels very hopeful.

"Now, we want to be cautious, of course, and we don't want to say that this is the cure for tomorrow or the next day for everybody," he said. "At the same time, what we've observed is really convincing, and we just hope that we can move this along to make it available to patients."

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Sickle cell anemia patient 'cured' by gene therapy, doctors say - CNN

Gene Therapy Shows Promise for Aggressive Lymphoma – WebMD

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By Margaret Farley Steele

HealthDay Reporter

TUESDAY, Feb. 28, 2017 (HealthDay News) -- An experimental gene therapy for aggressive non-Hodgkin lymphoma beat back more than a third of cancers that seemed untreatable, the therapy's developers report.

Thirty-six percent of over 100 very ill lymphoma patients appeared disease-free six months after a single treatment, according to results released by the treatment's maker, Kite Pharma of Santa Monica, Calif.

These patients had not responded to usual treatments and had no other options, Kite said Tuesday in a news release.

Overall, more than four out of five patients with the blood cancer saw their cancer reduced by more than half for at least part of the study, the company said.

"This seems extraordinary ... extremely encouraging," one cancer specialist, Dr. Roy Herbst, told the Associated Press.

But Herbst, who is chief of medical oncology at Yale Cancer Center in New Haven, Conn., said longer follow-up is needed to see if the benefit continues.

Still, he said, "This certainly is something I would want to have available." Side effects, which had been a concern, seemed manageable in this study, he said.

The therapy -- called CAR-T cell therapy -- enables the patient's own blood cells to kill the cancer cells.

Lymphoma is a general term for cancers that begin in the lymph system. The lymph system is part of the immune system, which helps the body fight disease.

Here's how the treatment works: A patient's blood is filtered so immune cells called T-cells can be altered to contain a cancer-fighting gene. The cells are returned to the patient intravenously, and the cancer-targeting cells then multiply in the patient's body.

The U.S. National Cancer Institute developed the gene approach and licensed it to Kite. Now, Kite and another pharmaceutical giant, Novartis AG, are competing to gain approval of the treatment, according to the AP.

Kite reportedly intends to seek U.S. Food and Drug Administration approval this spring and approval in Europe later this year. It could be the first gene therapy approved in the United States, the news report noted.

Although the therapy appears to benefit a significant number of patients, it is not risk-free. Researchers believe two patients died of treatment-related causes, the AP reported.

Other side effects included anemia or other blood problems that were treated, and neurological problems such as sleepiness, confusion, tremor or difficulty speaking, which typically lasted only a few days, the wire service reported.

Overall, however, the therapy seems safe, according to Dr. Steven Rosenberg, chief of surgery branch at the National Cancer Institute. He was not involved with the study.

"It's a safe treatment, certainly a lot safer than having progressive lymphoma," Rosenberg told the AP. He said he has a patient who was treated this way who is still in remission seven years later.

The cost of such treatment hasn't been reported yet, but immune system therapies tend to be very expensive.

The results are scheduled for presentation at the American Association for Cancer Research conference in April. Until published in a peer-reviewed medical journal, the data and conclusions should be considered preliminary.

WebMD News from HealthDay

SOURCES: Associated Press, Feb. 28, 2017; Kite Pharma Inc., news release, Feb. 28, 2017

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Gene Therapy Shows Promise for Aggressive Lymphoma - WebMD

In world-first breakthrough, French doctors use gene therapy to treat rare blood disease – RFI

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In world-first breakthrough, French doctors use gene therapy to treat rare blood disease - RFI

Gene therapy offers hope for newborns with severe immune disorder – University of California

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Infants born with a type of the devastating immune disorder SCID, or bubble boy disease, may have the option of a novel gene therapy treatment, thanks to a clinical trial atUCSF Benioff Childrens Hospital San Francisco.

The trial is funded by a five-year, $11.9-million grant from theCalifornia Institute for Regenerative Medicine (CIRM)to test technology developed by St. Jude Childrens Research Hospital that delivers a functional gene into the patients blood-producing stem cells. If successful, the gene therapy could provide an alternative to stem cell transplants using donor cells, which can result in serious infection.

The trial expects to treat up to 15 children over the next five years and is open to patients with X-linked severe combined immunodeficiency disease (X-linked SCID), which affects only males. This is the most common form of SCID, which occurs in 1 in every 60,000 newborns, and is caused by defects in the functioning of lymphocytes the white blood cells that are the advanced fighting forces of the immune system. Babies born with SCID appear normal at birth but become sick from infections, skin rashes and failure to gain weight at 3-to-6 months of age. Without a stem cell transplant, they may die before their first birthday.

What is unique about this trial is that the patients own bone marrow stem cells are collected and corrected with the gene therapy, and the corrected cells are then reinfused into the patient, saidMorton Cowan, M.D., of theUCSF Division of Allergy, Immunology, and Blood and Marrow Transplant, and principal investigator of the trial at UCSF.

In stem cell transplants from a donor other than the patient, up to 20 percent of patients with SCID will develop graft-versus-host disease, in which the donor cells attack the recipients tissues. In addition, there is always a risk of the recipient rejecting the donor cells, Cowan said. Using the patients own stem cells means no rejection and no graft-versus-host disease.

The bone marrow transplant program at UCSF is among the largest SCID transplant centers in North America. UCSF pediatric immunologistJennifer Puck, M.D., is known for pioneering the SCID screening method and for nominating SCID to a federal advisory committee for inclusion in the newborn screening panel. Since the screen became available in California in 2010, UCSF has treated more than 30 infants diagnosed with SCID by newborn screening.

UCSF also played an instrumental role in the St. Jude treatment protocol by including a targeted chemotherapy agent, busulfan, along with the gene therapy, which is expected to optimize immune correction. While previous trials have tested gene therapy for this condition, they did not combine it with chemotherapy and had only partial immune correction. Since a low dose of the medication is used, short- and long-term effects are expected to be minimized.

Three patients already have been treated with this lentiviral gene therapy vector two at St. Jude and one at UCSF. The transduction process, in which genetic material is transferred via vector, currently takes place at St. Jude, which freezes the transduced cells and returns them to UCSF for infusion into the patient. The CIRM funding will enable UCSF to begin doing transductions using the St. Jude vector at theUCSF Pediatric Cell Therapy Laboratory, as well as covering the cost of treating patients in the trial.

We believe this trial will not only help us understand more about how lentiviral gene therapy works, but how the use of low-dose busulfan potentially will be effective in treating other non-malignant diseases like sickle-cell anemia, chronic granulomatous disease, marrow failure syndromes and even some cancers in which the patient is too ill to undergo the more toxic traditional treatments, said Cowan.

It will also give us a better idea of what toxicities may be associated with the use of these new vectors, in particular whether they are indeed safer than the older, gamma-retroviral vectors that were associated with a high risk of leukemia, seen in early gene therapy trials for X-linked SCID and other primary immune deficiencies.

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Gene therapy offers hope for newborns with severe immune disorder - University of California

Bluebird’s star gene therapy patient still doing well after 15 months-plus; Takeda, Arix partner on biotech startups – Endpoints News

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Every biotech enjoys a good success story, and bluebird bio $BLUE got their writeup in the New England Journal of Medicine. Patient 1204 in bluebirds program for LentiGlobin gene therapy program has had more than 15 months of followup, and his physicians say that the teenager has been free of the painful symptoms of sickle cell disease, able to lead a normal life and stop painful transfusions. Thats all a positive, but bluebirds gene therapy has also proven to be somewhat variable among patients. One success story does not make for a successful therapy, but its still a plus.

Right on the heels of raising 100 million in an IPO, the UKs Arix has struck a deal to partner with Takeda in fostering new biotech startups. The Japanese company has been scaling way back on R&D in the country as Takeda revamps its approach to drug development.

You make hay while the sun shines, and you sell stock right after good data arrives. So its not a big surprise to see Kite line up a sale of 4.75 million shares at $75.00. Its stock $KITE, buoyed by recent 6-month data on its lead CAR-T, is down 5% in morning trading.

The UKs Adaptimmune has opened up its US headquarters in Philadelphias Navy Yard. The cancer company has 92 staffers in the area and plans to boost that figure to 120. Adaptimmune is closely tied to GSK, which also has extensive R&D operations in the area.

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Bluebird's star gene therapy patient still doing well after 15 months-plus; Takeda, Arix partner on biotech startups - Endpoints News

How the Italian Futurists shaped the aesthetics of modernity in the … – The Conversation UK

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Visions of the future, from the early 20th century.

This article is based around a transcript of a segment from The Anthill 10: The Future, a podcast from The Conversation. Gemma Ware, society editor at The Conversation and a producer of The Anthill, interviewed Selena Daly, an expert on the Italian Futurists.

When the Italian journalist Filippo Tommaso Marinetti went off to the frontlines of World War I, he was thrilled to be pedalling there on a bicycle. Back in 1915, bikes were an avant-garde mode of transport and Marinetti was an avant-garde kind of guy. Hed made waves across Europe a few years earlier when he launched the Futurist Manifesto.

Selena Daly: Marinetti, who was a master at advertising and self-promotion, got the first manifesto published on the front page of the Paris daily newspaper Le Figaro in February of 1909. This really was a very bold launch of an artistic and cultural movement at this time and got a lot of attention also around the world.

Selena Daly is a lecturer in Italian studies at University College Dublin and an expert in the Italian Futurists. Marinettis vision of the future was built around high praise for technology and the aesthetics of modernity.

SD: So he praised in this manifesto the speeding automobile, steamships, locomotives. All of these technologies that perhaps to our eyes now may seem a little bit quaint but at that time were really at the cutting edge of technology. So very famously, Marinetti in that manifesto praised the speeding automobile as being more beautiful than the famous Greek sculpture the Winged Victory of Samothrace which stands in the Louvre then and still today.

It was a movement that began with literature and poetry and spread to sculpture, fine art, music and even textiles. For example, this 1921 piece called Fox-trot Futurist by an Italian composer, Virgilio Mortari, was influenced by the Futurists. Marinettis vision was as destructive and provocative as it was creative and forward-thinking.

SD: He felt that Italy as a country was completely weighed down by the baggage of the Renaissance and the baggage of ancient Rome and its classical past. And he really wanted Italy to just stop looking backwards always and instead look to what the future could offer them in terms of inspiration for art and literature. And in that first manifesto he says he wants to rejuvenate Italy which he found very stagnant and therefore he said that everyone should set fire to the libraries, flood the museums and in this way break all links with the past.

With World War I in the offing, Marinetti and his band of followers quickly agitated for Italy to join the fight. They felt that war would help bring their Futuristic vision into being.

SD: One of the most famous slogans that Marinetti coined was in that very first manifesto where he said that he praised war as the sole hygiene of the world. The idea there should be a purging war which would rid Italy and Europe of all of its obsession with the past and they could move forward to a brighter future.

It took nine months for Italys leaders to agree to join the war during which time the Futurists campaigned vigorously for intervention. When Italy did enter the war on the side of the Allies in May 1915, Marinetti and his group of fellow Futurists signed up as soon as they could.

SD: They were terribly excited by the bombardments. They found this to be an inspiration also for their art and in very many ways putting into practice what they had preached and what they had thought about and imagined in advance of World War I.

When the war ended in 1918, the Futurists went through an intense period of political engagement, forming the Futurist Political Party and forming a close alliance with Benito Mussolini and his Fascist movement. The Futurist party wanted to make Italy great again. They wanted a country that was no longer in servitude to its past where the only religion was the religion of tomorrow. Their manifesto promised revolutionary nationalism, and included ideas such as totally abolishing the senate and the gradual dissolution of the institution of marriage. A 1914 design by futurist architect Antonio Sant'Elia. Antonio Sant'Elia

SD: But in the end of 1919 there were Italian elections and the Futurists and the Fascists performed disastrously. So they received less than 2% of the vote in Milan and its at that point that Marinetti actually decides that parliamentary politics isnt for him and he withdraws. He disbands the Futurist political party and he withdraws completely from parliamentary politics because he feels disillusioned and he feels that the message that he has isnt getting through.

Post-1920, Futurism no longer goes down the parliamentary politics route but it was, after 1924, very closely aligned with Mussolinis Fascist movement. So while they may not have been engaged in parliamentary parties they were very much on the side of the Fascist regime and that didnt change at all during Marinettis lifetime.

Marinettis association with Fascism has tainted the Futurists legacy ever since.

SD: Obviously some Futurists distanced themselves from the movement because of this alignment with Fascism. But others didnt. Its interesting a lot of the art in the 1930s and some of the 1940s is what can be described as Fascist pro-regime art. There are a lot of portraits of Mussolini done in a Futurist style for example. And the Futurists, while they were never the official state art of Fascism because Mussolini never wanted to proclaim one art to be the state art of Fascism the Futurists were still featured at official events and did have this very strong alignment with Musssoinis regime at that time.

Marinettis allegiance to Mussolini went right up to his death in 1944 in Bellagio in the north of Italy, near to the puppet regime run by Mussolini towards the end of World War II.

SD: Because there was such a cult of personality also around Marinetti and he was really the focal point of the entire movement it did rather peter out at that stage after his death and then at the end of the war as well. So there were surviving Futurists who did try in the 1940s and 1950s to keep Futurism alive and there was an interest in Futurism most definitely, but it was tainted by Fascism and there was a reluctance in many circles to really address the Futurist art and Futurist literature on its merits because of the shadow of Fascism that was hanging over it.

Italys relationship with Futurism is still complicated, but some Futurist images have remained iconic.

SD: There is a sculpture of Boccioni, one of the most famous Futurist artists, actually featured on the Italian Euro 20 cents coin, just to give an indication of how important the Futurist aesthetic is to a vision of modern Italy today. Boccioni, died actually in 1916. He died under arms, he actually fell off his horse in training so he didnt have the glory of a battlefield death that he may have wished for because he was also very belligerent.

But he was never tainted by Fascism because he died before Fascism actually came into being. So therefore its much easier to place a Boccioni sculpture on a Euro coin in Italy because he doesnt really have those other connotations and other associations with Fascism.

And the Futurists did help shape the way others in the 20th century went on to imagine what the future could look like.

SD: The Futurist aesthetic had a very profound influence on the language of advertising for example in the 20th century. For example, BMW recently said that they were very much influenced by the Futurist aesthetic in the design of one of their cars. There are fashion houses that are still using Futurist prints and Futurist textiles to inspire their collections. There is still an affinity for the Futurist aesthetic even today.

So while Marinettis technological, streamlined vision of the future may have been born out of a specific political moment, it has continued to resonate. Even the generic use of the word Futurist today remains strongly connected to Marinettis vision from 1909.

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‘Technical futurist’ will provide manufacturing conference keynote address – Herald-Whig

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Posted: Mar. 2, 2017 11:30 am

QUINCY -- John McElligott says what he does for a living will one day be considered commonplace rather than futuristic.

McElligott is a self-described "technical futurist," someone who "reads all the different trends that are coming together" and how they apply and can help various industries.

The trends that McElligott deals with are centered on the potential impacts of "machine learning and artificial intelligence."

McElligott will deliver the keynote address for the inaugural Tri-State Manufacturing Conference for Illinois, Iowa and Missouri on March 15 at John Wood Community College.

McElligott is founder and CEO of York Exponential based in York, Pa. The company develops and leverages machine learning and artificial intelligence and produces and integrates collaborative robots designed to work alongside human workers in manufacturing.

"Embracing Disruption" will be the topic of McElligott's keynote speech, which will serve as a crash course about the opportunities to be found in emerging technologies that embrace robotics. McElligott will try to help manufacturers, related businesses and communities decipher the impact innovations will have on the future.

"All companies are soon going to have a (technical futurist), even though the position might go by another name," McElligott said.

He said emerging technology will soon be changing the face of industry by the month rather than by the year.

"Companies, even smaller ones, have to be able to understand trends and exponential growth," he said.

McElligott has worked extensively in what he calls "third-tier cities," those similar to Quincy with populations of about 40,000. He believes in the power of community networking through communication and technology and is a national speaker on exponential technology, robotics, artificial intelligence, economic development and disruption.

The conference will feature breakout sessions on supply chain/logistics, the talent pipeline, sales growth, market intelligence, technology adoption and leadership.

People attending the conference will have the opportunity to network and take part in a small-group discussion to learn how some manufacturers apply new technology in daily operations. Vendors also will display new technology and products.

The conference will take place 8 a.m. to 4 p.m. at JWCC's Heath Center.

Cost for the conference is $45, including lunch. The event is open to leaders and employees from small- and large-size manufacturers, plus suppliers and related businesses in the industry. Registration details and more information are available at jwcc.edu/tristatemfg, or by contacting JWCC at 217-641-4971 or lewis@jwcc.edu.

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Businesses Need Futurism to Stay Ahead – Business.com

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How futurism is becoming the newest business strategy and why it's important.

The technology market has been rapidly changing due to futurist ideas. Companies are investing money into technology that will safeguard their businesses for the future. Large businesses cannot prevent issues coming up in the near future, but hiring futurist experts will mitigate what is to come for them.

In 1980, Edward Cornish of the World Future Society gave some groundbreaking predictions. He made 29 predictions of what the future will look like, and 11 of them became reality. One of the first predictions he made was that there would be artificial hearts available for transplants, which came true a few years later. He predicted that the construction industry would mostly rely on automated machinery with minimal labor. Unfortunately, he also predicted that governments will struggle to distribute food and labor for the majority of workers.

Other experts in futurism agree that rapid technology changes are ruining certain businesses. Jobs are being lost around the world, and governments do not know how to cope with these changes. Both businesses and governments will need to study futurism in order to implement changes that safeguard their economies.

HR departments have been recruiting futurist experts to improve the efficiency of their departments. Futurists aid them in determining new skill sets that departments should be looking for to gain an edge in their industries.

Organizations like the Rockwell International Corporation have appointed their own teams to assess their goals of the future. These teams must analyze the climate of the market to determine what moves the company must take to still be alive in 10 years. The team will also assess multiple paths for the company to take for the predictions of many alternative futures.

Even small businesses can take advantage of following futurist ideas. Long-term trends will significantly impact the demand for small shops or services, and many businesses may even become irrelevant. When new technology is introduced to the marketplace, business owners should assess how they can take advantage of the new trend. If a new demographic is moving into certain neighborhoods, the local shops must conform to appeal to them. These are just a few basic examples of futurism.

Entrepreneurs of small businesses should focus on imagining what the customer will need in the near future. Focusing on small, short-term niches will assure a collapse of businesses after the niche dries up.

Becoming personally invested in a product is also a bad idea. Many products may not even meet the demands of the public. A good example is the hordes of forgotten projects on websites like Kickstarter.

Even simple gadget trends are changing the way technology businesses are going. The internet of things has made many companies shift toward internet connectivity in their devices. Refrigerators, video game consoles, televisions, doors, lights, toasters, ovens, coffee machines and other appliances have been conforming to IoT trends.

On the other hand, there is an overinvestment in certain technology gimmicks that draw away from more important things. Pagers were an obsession during the 1990s, but the market should have focused on expanding mobile phones instead. Answering machines are another device that was quickly made irrelevant due to the rapid changes in phone technology.

Hotels have also been adapting to the way the internet shifts the market. Online booking is practically a must, since the majority of bookers are finding the hotel from the internet. Airbnb certainly changes accommodation prices in many tourist areas, and prices may shift down or up. The future of travel accommodation is shifting toward alternative means, so hotels must figure out a way to mitigate this.

Technology marches on, and the needs of consumers will keep changing with it. Businesses should be doing everything they can to future-proof themselves as much as possible.

Image from Michael R. Ross/Shutterstock

Jason Hope

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In Case You Missed it, The Blockchain Revolution’s Officially Begun – Futurism

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Blockchain: More Than Bitcoin

Blockchain and cryptocurrency are relatively new. Most people might even think that Bitcoin invented just in 2009 and probably the most popular blockchain-based cryptocurrency out there is the only one of its kind. But blockchain is more than just Bitcoin.

Blockchain is a digital ledger of transactions. Its public and is not governed by a central body. As such, theres a relative level of transparency coupled with security through cryptography. In other words, its safe and reliable, and monitored by hundreds of miners who keep these ledgers. Its quickly becoming of interest to not just existing financial markets, buthumanitarian and sustainability efforts.

At the moment, blockchains are most frequentlyused for cryptocurrencies like Bitcoin. But it has other uses like what distributed public blockchain network Ethereum does. Instead of focusing on just digital money like Bitcoin, the Ethereum blockchain runs the programming code of decentralized applications, allowing for enterprise use. Transactions in the Ethereum network rely on a crypto-token (also known as security tokens) called Ether.

While theres specific no research lab dedicated to blockchain and cryptocurrency just yet, Hong Kong-based cryptocurrency research and development company IOHK wants to change that. IOHK was established in 2015 by Jeremy Wood and Charles Hoskinson, one of the founders of Ethereum. Theyre planning toinvest up to $1 million in two facilities for research: one at the University of Edinburgh, and the other Tokyo Institute of Technology.

The labs will cover topics such as cryptography, smart contracts, and how to upgrade cryptocurrency systems. Best of all, their research will be open source and accessible to everyone. This is commonly not done in the startup setting, Hoskinson told Business Insider. Usually, this is something you do if youre a company like Microsoft Microsoft has research campuses at many major universities.

Hoskinsonalso said that setting up these research labs can provide perspective and better understanding of the growing blockchain technology. After having discussions, they [the experts] said, actually we dont have answers to a lot of these fundamental questions, explained Hoskinson. We said, how do we get those answers? And they said, we need to write some papers, we need to do some basic research. Over time we started moving into the university research space.

We already know that blockchain is more than Bitcoin, but now that there will be research labs dedicated to understanding its potential, the future of the technology is bound to develop rapidly. The days of digital cash becoming globally dominant could arrive sooner than we think.

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Scientists Have Made a Huge Breakthrough In Cryogenics – Futurism

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Cryopreservation

Cryopreservation is the process of freezing organs and tissues at very low temperatures in order to preserve them. While it sounds simple in theory, only a handful of cells and tissues have survivedthis method. This is because while science has successfully developed ways to cool organs to the very low temperatures required for preservation, thawing them out has proven far more difficult. As the specimen thaws, itforms ice crystals, which can damage the tissue and render organs unusable.

Right now, the process is only a viable option for small samples, such as sperm or embryos. Previous efforts using slow warming techniques have proven to be effective on samples of that size, but havent worked forlarger tissue samples, like whole human organs. The inability to safely thaw the tissue has also precluded the theoretical concept of cryogenically preservingentire human bodies, with the intention of reanimating them later. The concept has roots in cryogenic technology, but is actually referred to as cryonics, and the scientific community generally considers it to be more science fiction than science fact at least for the time being.

A recentstudy has made a significant breakthrough which may well begin closing that gap even more. Using a new technique, scientists were able to cryopreserve human and pig samples, then successfully rewarm it without causing any damage to the tissue.

As lead researcher John Bischof from the University of Minnesota notes:

This is the first time that anyone has been able to scale up to a larger biological system and demonstrate successful, fast, and uniform warming of hundreds of degrees Celsius per minute of preserved tissue without damaging the tissue.

By using nanoparticles to heat the tissues at an equal rate, scientists were able to prevent the formation ofthose destructive ice crystals. The researchers mixed silica-coated iron oxide nanoparticles in a solution and applied an external magnetic field to generate heat. The process was tested on several human and pig tissue samples, and it showed that nanowarming achieves the same speed of thawing as the use of traditional convection techniques.

One theoretical application for this discovery would be, of course, bringing cryogenic life-extension techniques out of the realm of science fiction and into reality. But were not quite there yet.

A more practical application for the technique wouldbeto safely preserve and store organs for extended periods, thus improving the logistical challenges behind organ transplantation.

According to statisticsfrom the United Network for Organ Sharing, 22 people die every dayin the US while waiting for organ transplants. Contrary to popular belief, this isnt because there is a shortage of organs being donated its because organs cannot be preserved for more than a few hours. So, while there are available organs ready to be transplanted, the time it takes to find a matching recipient and transport the organ safely to their location often exceeds the window of time in which the organ remains viable for transplant.

Over half of donated hearts and lungs are thrown out each year because they dont make it to patients in time. They can only be kept on ice for four hours, and while some organs can last longer than others without a blood supply during transport, its still not a longenough in many cases.

If only half of these discarded organs were transplanted, then it has been estimated that wait lists for these organs could be extinguished within two to three years, Bischof adds. With the help of cryopreservation technology, we may be well on our way to keeping donated organs viable for longer meaning they could be transported to patients who need them even if distance and time stands between them.

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Tesla’s Cars Will Soon Be Just As Affordable as Gas-Powered Vehicles – Futurism

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In Brief

Part of Elon Musks grand plan of making the world a greener place and addressing the damage caused by climate change is ensuring that people have access to renewable technology. This means both introducing the technology and bringing the cost down so that people can actually have viable alternatives to traditional, carbon-emitting sources.

Teslas Gigafactory was a big part of this plan. Once the factory became operational, Tesla was gunning to reduce their battery cost by 30 percent. Now, a recent announcement from the company hints that it might be possible to bring it down even more. In a promotional video displayed in some stores, it seems that the factory was able to achieve a 35 percent reduction in battery cost.

No numbers have been officially released, but given the small bits of information that have gradually come outto the public, it seems that Tesla is definitely getting closer to that $35,000 price tag that the company is targeting for its Model 3.

Since 2001, battery cost for electric vehicles (EVs) has been reduced by80 percent. Even so, the current cost of todays long-range EVs is still not very affordable for most people. To be able to bring down the overall cost, the priceof battery cells and packs must significantly be lowered.

Early in 2016, Tesla said that price point was already below $190/kWh, prior to the Gigafactory starting production. With the factory now up and running, and the Tesla/Panasonic partnership already starting production for the 2170 battery cell, they could be able pull down the cost to $125/kWh.

The goal would be to reach $100/kWh. At this rate, EVs will be on par with gas-powered vehicles and chances are, people will be more likely to adopt the renewable technology if they can do so without having to spend more money.

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The ISS Is Getting Its First African-American Crew Member in 2018 – Futurism

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The Remarkable ISS

The International Space Station (ISS) may be an impressive technological marvel, but its also tangible proof of what humans can accomplish when we set aside political, religious, gender, or racial differences and focus on science.Built by Russia and funded by the United States, the $100 billion space station is the result of more than a dozen countries working together.

A remarkable amount of effort went into the successful creation of the ISS, which has now been in operation for more than 16 years. It currently orbits Earth at an altitude of 354 kilometers (220 miles), traveling at 28,163 kilometers per hour (17,500 miles per hour). The space station orbits our planet every 90 minutes, and an acre of solar panels keep the outpost running.

Right now, the ISS is home to several crew members from various nations, all of whom are focused on learning about how humans can live and work in space.It is arguably the most visible example of international cooperation and everything that can be achieved when nations collaborate.

Soon, a new addition to an ISS expedition crew will make history aboard the space station. When Jeanette Epps joins Expedition 56 in March 2018 as a flight engineer, she will become the first African-American to join the ISS as a crew member.

Epps holds a doctorate in aerospace engineering from the University of Maryland and served as a fellow in NASAs Graduate Student Researchers Project, an initiative that hopes to increase engagement amongst students who want to pursue advanced degrees in science, technology, engineering, and mathematics (STEM) fields.

In a recent interview with New York Magazine, Epps shared her thoughts on joining the ISS:

There have been three African-Americans who have visited ISS, but they havent done the long-duration mission that I am undertaking. Ill be the one spending the longest time on the ISS. As a steward, I want to do well with this honor. I want to make sure that young people know that this didnt happen overnight. There was a lot of work involved, and a lot of commitment and consistency. It is a daunting task to take on.

While Epps will be the first African-American to board the ISS for a long-term expedition, numerous African-American women have lent their expertise to the success of NASA. As far back as the 1950s, African-American women were contributing to humanitys mission to explore the unknown, and soon, Epps will be able to add her name to the list of people breaking new ground in space exploration.

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The ISS Is Getting Its First African-American Crew Member in 2018 - Futurism

China Is Replacing 70000 Taxis With Electric Cars – Futurism

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The Future of Taxis

Electric vehicles have been growing in popularity among fleet operators, and soon, Beijing may find itself earning a reputation as the hub of the electric taxi. The Chinese city is home to one of the most important taxi fleets in the world, numbering around 70,000, and under a new program for air pollution control that will begin implementation this year, those taxis will be going electric.

According to a report by National Business Daily, the transition to electric cars will cover all new taxis registered in the region. All newly added or replaced taxis in the city of Beijing will be converted from gasoline to electricity, according to a draft work program on air pollution control for Beijing, Tianjin, Hebei, and surrounding areas in 2017, the report reads.

The city isnt the first to push for electric taxis. Previously, Shenzhen and Taiyuan both announced similar policies for their taxis, but the move is significant for a municipality of over 20 million people.

The total fleet conversion is expected to cost around 9 billion yuan ($1.3 billion USD), but the cost is worth it. Electric cars will potentially save taxi companies from gas expenses and could lower maintenance costs. They will also help save the city from pollution, as Beijings populace is exposed to hazardous air quality on a semi-regular basis.

The move to electric cars isnt cheap, though, which is why Liu Jinliang, chairman of Geelys ride-hailing arm Caocao, hopes the government will provide subsidies to taxi companies. National Passenger Cars Association secretary-general Cui Dongshualso hopes that the government will speed up the construction of charging facilities.

This new mandate, coupled with the Chinese governments recentrelaxation of restrictions on car manufacturingin an attempt to draw more electric vehicles, demonstrates the countrys seriousness in investing in electric cars. According to Electrek, the nationis now the worlds biggest electric vehicle market, with a fleet of over 600,000 electric cars more than both the U.S. and European markets combined. Soon, theyll be adding another 70,000 to that total.

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Doctors Claim They’ve Cured a Boy of a Painful Blood Disorder Using Gene Therapy – Futurism

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Potential Treatment

Gene therapy has been available for quite some time now. Advances in modern medical science, particularly in stem cell research, have made it possible to use DNA to compensate for malfunctioning genes in humans. The therapies haveeven proven effective fortreating rare forms of diseases. Now, a research team in France has shown that gene therapy may be used to cure one of the most common genetic diseases in the world.

The team, led by Marina Cavazzana at the Necker Childrens Hospital in Paris, conducted stem cell treatment on a teenage boy with sickle cell disease. The disease alters theblood through beta-globin mutations, which cause abnormalities in the blood proteinhemoglobin. These abnormalities cause the blood cells (which have an irregular shape, like a sickle, hence their name) to clump together. Patients with sickle cell disease usually need transfusions to clear the blockages their cells cause, and some are able to have bone marrow transplants. About 5 percent of the global population has sickle cell disease,according to the WHO. In the United States alone, the CDC reports that approximately 100,000 people have sickle cell disease.

The patient is now 15 years old and free of all previous medication, Cavazzana saidwhen discussing the outcome of their study. He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers. Their research is published in the the New England Journal of Medicine.

The particular treatment given to the teenage boy at Necker Childrens Hospitalbegan when he was 13 years old. The team took bone marrow stem cells from the boy and added mutated versions of the gene that codes for beta-globin before putting these stem cells back into the boys body. The mutated genes were designed to stop hemoglobin from clumping together and blocking blood vessels the hallmark of sickle cell disease.

Two years later, the boys outcomelooks promising.All the tests we performed on his blood show that hes been cured, but more certainty can only come from long-term follow-up, Cavazzan said. Her team also treated seven other patients who also showed promising progress.

If the method shows success in larger scale clinical trials, it could be a game changer, saidDeborah Gill at the University of Oxford, The fact the team has a patient with real clinical benefit, and biological markers to prove it, is a very big deal.

Other research involving gene therapy is also showing similar promise. One which has already been approved by the FDA is a potential treatment for blindness. Others look at treating Parkinsons disease or evenprolonging human life. What these studies show is that gene therapyand stem cells may be able togive hope to patients with diseases that have long been considered incurable.

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Paul Ryan’s Misguided Sense of Freedom – New York Times

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New York Times
Paul Ryan's Misguided Sense of Freedom
New York Times
He went on to argue that Obamacare abridges this freedom by telling you what to buy. But his first thought offers a meaningful and powerful definition of freedom. Conservatives are typically proponents of negative liberty: the freedom from constraints ...

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NRB head: Islam, homosexuality trumping Christians’ freedom – OneNewsNow

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The host and leader of the National Religious Broadcasters (NRB) Christian Media Convention in Orlando, Florida, impressed to attendees that Islam and homosexuality are increasingly undermining the constitutional rights of Christians throughout America.

NRB President Jerry Johnson began this weeks convention in the Sunshine State by talking about the three major areas of his organizations ministry.

"Our mission is threefold: Advancing biblical truth, promoting media excellence and defending free speech," Johnson proclaimed Monday night, according to The Christian Post (CP). "Religion, that's NRB. Speech, that's NRB. Press, that's NRB. I don't know another group that does all three. Some do religion, some do speech and some do press."

Johnson then turned to the foundational document from which Americans liberties are derived a document he dubbed as Americas birth certificate, more commonly known as the Declaration of Independence, which he cited:

"We hold these truths to be self-evident, that all men are created equal, that they are endowed by their Creator with certain unalienable rights, that among these are life, liberty and the pursuit of happiness," Johnson quoted from the 241-year-old document.

However, NRB went back another 250-plus years to commemorate the freedoms that Americans still hold dear five centuries later by tapping into famous theologians and authors to address the crowd.

NRB is celebrating the 500th anniversary of the Protestant Reformation at this year's convention, which is featuring such speakers as Devon Franklin, R.C. Sproul, Lee Strobel and Eric Metaxas, CPs Jeannie Law reported from the Florida conference. The theme verse for NRB's Proclaim 17 conference comes from Leviticus 25:10, which says, Proclaim liberty throughout the land to all its inhabitants."

Homosexuality and Islam challenging Christianity

The Christian leader then warned Christian journalists, broadcasters, leaders and other communicators within the media world that there are two threats believers in America must take very seriously.

"We speak frankly here, the liberty and the freedom [part] is being challenged for us as broadcasters on two fronts sexuality and Islam," Johnson told the gathering of Christian communicators.

Addressing the LGBT agenda

Johnson went on to remind the Christians in attendance how their and their audiences traditional values have been trampled to accommodate the increasingly influential politically correct LGBT agenda.

"For now, the U.S. government has decided that same-sex marriage is legal, Johnson recounted to crowd. President Donald Trump also recently endorsed the legalization when he told CBS' 60 Minutes that these cases have gone to the Supreme Court; they've been settled and he's fine with that."

The leader of NRB then talked about why the legalization of same-sex marriage poses major obstacles for Christians in the workplace.

[The problem is that Christian artists shouldnt be forced to] make the cake, provide flowers, do the pictures and sing the songs," Johnson stressed before highlighting several NRB guests this year.

Former Atlanta Fire Chief Kelvin J. Cochran, who was terminated for expressing his biblical beliefs about homosexual behavior, was mentioned by the NRB head before he shared about the how the Benham Brothers were kept from hosting their own show on HGTV over some alleged remarks that were considered by some as being anti-gay.

"People are penalized for this," Johnson expressed in dismay while reflecting on the Christian mens prematurely ended careers.

The problem with Islam

Johnson also addressed how terrorism and violence are only part of the problem concerning Islam.

"But it's the intolerance It's the sharia It's the blasphemy codes," NRBs leader voiced to the Florida crowd. "Is there any country where the majority is Islam [and] it's safe for someone to convert from Islam to Christianity? Is there any culture dominated by this worldview, where you can critique Muhammad or the Quran? It is absolutely antithetic to freedom of speech, freedom of religion or freedom of the press, and we must be aware of this and alert to this."

The various ways in which Islam has infiltrated all parts of the glove to threaten the very values, freedoms and safety of people everywhere was then discussed.

"It is a fact in Canada and in the U.K., you can be censored, you can be fined [and] your license can be pulled," he stressed.

Continuing on the same note, Johnson shared how a member of the NRB was fined and had his license revoked north of the border for using the word Muslim instead of Islam during a program.

"I'd say the quickest way to lose freedom of speech is to not use freedom of speech, use-it-or-lose-it!" the media expert asserted. "We will make NRB for the First Amendment, what the NRA is for the Second Amendment."

He then emphasized how NRB will not back down to such bullying tactics that are geared to forward the politically correct agenda endorsing Islam in the media.

"If you're taking away these freedoms, NRB is a fight club and you better get used to it! Johnson exclaimed. Nixon wasn't paranoid they were really out to get him. Wake up. They are really out to get you.

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NRB head: Islam, homosexuality trumping Christians' freedom - OneNewsNow