Calif. has novel view of health care for undocumented …

Guadalupe Carrera, 36, with her daughter Eva Maqueda, 9, and son Jose Maqueda, 5, fills out an application for health insurance assistance at El Proyecto Del Barrio Family Health Care Clinic on Jan. 8, 2015.(Photo: Heidi de Marco, Kaiser Health News)

PASADENA, Calif. Angel Torres hasn't been to the doctor since coming to the United States illegally more than two decades ago. But now, his vision is getting blurry and he frequently feels tired. Torres, 51, worries he might have diabetes like his brothers.

"Time is passing," he said in Spanish. "I need to get checked out."

Torres is in luck. He lives in California, which has a dramatically different approach to health care for undocumented immigrants than most other states.

Several counties including Los Angeles, where Torres lives offer these immigrants free coverage at local clinics. In addition, as many as 500,000 low-income immigrant parents eligible for President Obama's new deportation relief likely will qualify for Medi-Cal, California's version of Medicaid. Already, young adults who were brought here as children have been granted similar immigration relief and can receive the state-funded insurance.

And in December, legislation was proposed to extend state-subsidized health insurance to everyone, including those barred from getting covered through the Affordable Care Act. Federal dollars could not be used.

The push to offer health insurance to all Californians regardless of immigration status is the latest in a series of immigrant-friendly state policies. Undocumented immigrants here can obtain licenses to practice medicine, law or other professions, and, as of this month, they can apply for driver's licenses.

There is no guarantee that other states will follow California's lead, but the size and demographic makeup of the state ensure it a prominent role in the national debate over covering undocumented immigrants.

If California "goes out on a limb it is a really big thing," said Randy Capps, director of research for the Migration Policy Institute, a nonpartisan think tank based in Washington, D.C.

California has been a leader on some immigration issues. For instance, it was one of the first states to offer in-state tuition to students here illegally. Now, as undocumented immigrants increasingly settle throughout the country, about 20 do so.

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MSF breathes life into Gokwe North health care system

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When Mdecins Sans Frontires (MSF) arrived in Gweru in 2004, in a joint operation with the Ministry of Health and Child Care, the effort brought amazing improvement in the citys health delivery system.

By Moses Mugugunyeki

Passengers push a truck along the muddy Nembudziya-Mashame road recently The poor road network has immensely contributed to the demise of the health delivery system in Gokwe North.

The people of Gweru were happy with the new health services brought to them. Seven years down the line however, it depressingly dawned on the people that what they had believed to be a life-long health menu, was in fact a temporary meal ticket.

While the government was aware that it was a temporary health delivery arrangement, it failed to capitalise on the availability of the health expertise to impart knowledge to local personnel to enable the continuation of such a health delivery system at the expiry of the joint venture.

So, when the MSF Gweru contract came to an end in 2011, the inadequacy was exposed. Serious challenges in the treatment of HIV and Aids and tuberculosis (TB), especially drug-resistant TB (DR-TB), resurfaced.

MSF, which had by then moved on to Gokwe North, apparently noticed the problem that had befallen Gweru and were determined not to let the same thing happen at their next port of call.

The strategy that we used in Gweru created gaps in the health delivery system after MSF had left, said Stambuli Kim, communications officer for MSF. Our objective in Gokwe North was then to reduce morbidity and mortality as a result of especially HIV and Aids and TB. We also ensured that activities would continue independently after MSF pulled out.

Zhomba Clinic

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MSF breathes life into Gokwe North health care system

Louis-Jeantet-Prize for Medicine goes to Emmanuelle Charpentier

Award honors fundamental research that is expected to be of considerable significance for medicine

IMAGE:This is professor Emmanuelle Charpentier, recipient of the Louis-Jeantet-Prize for Medicine 2015. view more

Credit: Helmholtz / Hallbauer&Fioretti

Prof Emmanuelle Charpentier from the Helmholtz Centre for Infection Research (HZI) in Braunschweig is one of the two recipients of the 2015 Louis-Jeantet-Prize for Medicine. The prize money of 700,000 Swiss francs is mostly attributed for the continuation of the awardees work. Charpentier receives the prize for harnessing an ancient immune defense system in bacteria - CRISPR-Cas9 - into a genome editing tool largely exploited by biologists and promising for curing human diseases.

Bacterial pathogens also possess an immune system that defends them against predators, and particularly viruses. When studying this system, Charpentier while she was a group leader at the Laboratory for Molecular Infection Medicine Sweden (MIMS) at Ume University and her team unravelled a unique mechanism - CRISPR-Cas9 - a pair of molecular scissors composed of a duplex of two RNAs linked to a protein. In collaboration with the team of her colleague Prof Jennifer Doudna, University of California, Berkeley, it was demonstrated that the mechanism could be harnessed into a powerful tool for genome engineering. The system is celebrated as a revolution for biology, and used by laboratories all over the world for different purposes.

"The CRISPR-Cas9 system has already breached boundaries and made genetic engineering much more versatile, efficient and easy", says Charpentier, who is the head of the department "Regulation in Infection Biology" at the HZI, Alexander von Humboldt Professor affiliated at the Hannover Medical School and a guest Professor at the Laboratory for Molecular Infection Medicine, Ume University, Sweden. The system can be used in various areas of biology and medicine and curing genetic disorders is only one possible application. The system also shows promises in areas like agriculture and the development of therapeutics for the or when it comes to developing treatment of chronical diseases such as HIV or cancer.

Established in 1986, the Louis-Jeantet-Prize for medicine has already been awarded to 82 researchers. Ten of the awardees subsequently won the Nobel Prize for Physiology or Medicine, or the Nobel Prize for Chemistry. As one of the best-endowed awards in Europe, the Louis-Jeantet Prize for Medicine fosters scientific excellence. It finances the continuation of innovative research projects of more or less immediate practical significance for the treatment of diseases rather than honouring completed work.

"I feel extremely honoured receiving this prestigious award", says Charpentier. "I see it as a motivation for my team to continue our work and will use the prize money to conduct further research on the mechanisms governing the pathogenicity of Streptococcus pyogenes".

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Louis-Jeantet-Prize for Medicine goes to Emmanuelle Charpentier

Gene therapy-associated cancer incidence depends on vector design

Gene therapy is a promising strategy to correct hereditary disorders. The approach takes advantage of viral vectors to deliver a corrected version of the mutated gene. Adeno-associated virus (AAV) has many features that make it a favorable vector for gene therapy. In animal models, AAV-mediated gene delivery is generally regarded as safe and has demonstrated efficacy for some genetic diseases. However, a recent study reported an increase in liver cancer in mice after AAV gene therapy. A new publication in the Journal of Clinical Investigation reveals that AAV vector design influences the likelihood of developing cancer in the liver. Charles Venditti and colleagues at the National Institutes of Health looked for the development of hepatocellular carcinoma (HCC) in a large number of mice that had received AAV gene therapy. HCC was associated with the AAV vector integrating within a specific site in the genome and inducing expression of microRNAs and a retrotranposon. Moreover, AAV dose, the choice of enhancer/promoter, and timing of delivery all influenced the HCC incidence. The results of this study provide insight into features that should be considered when designing AAV vectors for gene therapy.

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TITLE:

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

AUTHOR CONTACT:

Charles Venditti National Institutes of Health, Bethesda, MD, USA Phone: 301-496-6213; Fax: 301-451-3853; E-mail: venditti@mail.nih.gov

View this article at: http://www.jci.org/articles/view/79213?key=0ff2890aa2b1c1b59bca

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Gene therapy-associated cancer incidence depends on vector design

NIH researchers tackle thorny side of gene therapy

Pre-clinical studies in mice reveal ways to reduce cancer risk with modified treatment

National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome Research Institute (NHGRI), part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation.

"Effective and safe gene therapies have the potential to dramatically reverse diseases that are life-threatening for affected children," said NHGRI Scientific Director Dan Kastner, M.D., Ph.D. "This study is an important step in developing gene therapies that can be safely used to benefit patients."

Toxic side effects actually are rarely observed by researchers who have designed gene therapies using an adeno-associated virus (AAV) as a vector to deliver the corrected gene to a specific point in the cell's DNA. AAVs are small viruses that infect humans but do not cause disease. A vector is a DNA molecule of AAV used as a vehicle to carry corrected genetic material into a cell. AAV viruses are uniquely suited for gene therapy applications.

But one prior study did find an association between AAV and the occurrence of liver cancer. The present research addresses this problem in gene therapy for an inherited disease in children called methylmalonic acidemia, or MMA.

For 10 years, NHGRI researchers have worked toward a gene therapy to treat MMA. The condition affects as many as 1 in 67,000 children born in the United States. Affected children are unable to properly metabolize certain amino acids consumed in their diet, which can damage a number of organs and lead to kidney failure. MMA patients also suffer from severe metabolic instability, failure to thrive, intellectual and physical disabilities, pancreatitis, anemia, seizures, vision loss and strokes. The most common therapy is a restrictive diet, but doctors must resort to dialysis or kidney or liver transplants when the disease progresses.

In prior MMA gene therapy studies, researchers showed that mice bred to develop the condition could be restored to health by AAV gene therapy injection shortly after birth. The mice in the study survived into adulthood and were free from the effects of MMA.

"The corrected gene delivered by AAV is the most effective therapy we have developed so far to treat MMA," said Charles Venditti, M.D., Ph.D., senior author and investigator in NHGRI's Genetic and Molecular Biology Branch. "However, we have identified an important safety parameter related to the AAV gene therapy in our mouse models that is critical to understand before we move to human patient trials."

Now, in a long-term follow-up of the treated mice -- after mice reached about two years of age -- the researchers documented a 50-70 percent higher occurrence of liver cancer in AAV-treated mice compared with a 10 percent liver cancer rate in untreated mice. Dr. Venditti's team determined that the AAV vector triggered the cancer.

The research team performed additional experiments to detect where in the mouse genome the AAV vector delivered the corrected gene and how that related to any cancer development. In many mice that developed liver cancer, the AAV vector targeted a region of the mouse genome called Rian, near a gene called Mir341 that codes for a microRNA molecule. MicroRNAs are small, non-coding RNA molecules involved in the regulation of gene expression. When the AAV was inserted near Mir341, the vector caused elevated expression of the gene, which the researchers believe contributed to the occurrence of liver cancer in the mice. The authors note that Mir341 is found in the mouse genome, however, it is not present in humans.

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NIH researchers tackle thorny side of gene therapy

Gene therapy biotech Spark Therapeutics sets terms for $88 million IPO

Spark Therapeutics, which is developing gene therapy treatments for retinal dystrophies and hematologic disorders, announced terms for its IPO on Tuesday. The Philadelphia, PA-based company plans to raise $88 million by offering 5.5 million shares at a price range of $15 to $17. At the midpoint of the proposed range, Spark Therapeutics would command a fully diluted market value of $378 million.

Spark Therapeutics, which was founded in 2013, plans to list on the NASDAQ under the symbol ONCE. J.P. Morgan and Credit Suisse are the joint bookrunners on the deal. It is expected to price during the week of January 26, 2015.

Investment Disclosure: The information and opinions expressed herein were prepared by Renaissance Capital's research analysts and do not constitute an offer to buy or sell any security. Renaissance Capital, the Renaissance IPO ETF (symbol: IPO) or the Global IPO Fund (symbol: IPOSX) , may have investments in securities of companies mentioned.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of The NASDAQ OMX Group, Inc.

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Center for Retinal and Ocular Therapy at Penn Expands Relationship with Spark Therapeutics to Develop Potential …

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Newswise PHILADELPHIA The University of Pennsylvania has announced an expanded relationship with Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating, genetic diseases. As part of the expanded relationship, which included both an exclusive license agreement to certain Penn-owned intellectual property rights and a clinical trial agreement, Penn will be one of the clinical sites for a clinical trial to evaluate the treatment of a rare genetic form of progressive blindness.

This expanded strategic relationship between the two organizations is representative of Penns strong commitment towards direct engagement with the private sector to advance promising technologies, said John Swartley, Associate Vice Provost for Research and Executive Director, Penn Center for Innovation (PCI). When Penn researchers on the cutting edge of their fields are able to partner effectively with innovators in the private sector it has the potential to accelerate the advancement of exciting new approaches for the treatment of disease. PCI serves as the University of Pennsylvanias commercialization center and actively works with the private sector to foster research and development collaborations leveraging Penn technology and research.

Spark today announced the start of Phase 1/2 clinical trial for patients with choroideremia (CHM), which will take place at The Childrens Hospital of Philadelphia and the University of Pennsylvania. CHM is a rare, genetic eye disorder that causes progressive vision loss, ultimately leading to complete blindness. CHM is characterized by deletions or mutations in the CHM gene. It is a degenerative eye disease which affects males that manifests in childhood as night blindness and a reduction in visual field, followed by progressive constriction of visual fields leading, ultimately, to blindness. There is currently no approved drug treatment for the disease.

The launch of this clinical trial is the latest facet of the ongoing partnership between Spark and Penn.Expanding upon an earlier collaboration around SPK-RPE65, in December of 2014, Spark and Penn, through PCI, entered into an exclusive license agreement to certain Penn-owned intellectual property rights, including assets related to the choroideremia program. As a part of the license agreement, Penn received equity shares in Spark and may receive additional milestone payments and royalties on net sales dependent on the success of the SPK-CHM program.

The new trial is designed to assess the safety and preliminary efficacy of sub-retinal administration of SPK-CHM. The investigators plan to enroll up to 10 patients afflicted with the CHM genetic mutation. In addition to evaluating safety, the trial will help define the dose required to achieve stable or improved visual function and identify appropriate endpoints for subsequent clinical trials. The trial will build on the work of the clinical trial teams that have conducted trials of Sparks therapy known as SPK-RPE65, which has been observed in clinical trials to improve vision in patients with rare blinding conditions due to mutations in the RPE65 gene.

I have a particular interest in choroideremia, says Jean Bennett, MD, PhD, the F.M. Kirby Professor of Ophthalmology and director of the Center for Retinal and Ocular Therapy (CAROT) at the Perelman School of Medicine at the University of Pennsylvania, and one of Sparks scientific co-founders. I am thrilled to now be able to test our gene therapy treatments with the potential to help the many men living with this disorder.

"Editors note: The University of Pennsylvania has licensed technology involved in this research to Spark. Dr. Bennett is an inventor of this technology, and may benefit financially."

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Center for Retinal and Ocular Therapy at Penn Expands Relationship with Spark Therapeutics to Develop Potential ...

Futurist, Barbara Marx Hubbard: Conscious Evolution of Humanity – Video


Futurist, Barbara Marx Hubbard: Conscious Evolution of Humanity
For Beyond 50 #39;s "Emerging Trends" talks, listen to Futurist, Barbara Marx Hubbard. She #39;s known as a global ambassador for conscious change, co-founding the World Future Society and the Association ...

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SpaceX Raises $1 Billion In A Financing Round With Google And Fidelity

SpaceX today confirmed yesterdays reports that it has embarked on a new round of fundraising. The round includes two new investors, Google and Fidelity investments.

The total investment of the two companies is one billion dollars, which SpaceX says means that Google and Fidelity will collectively own just under 10% of the company. That gives the space startup a total valuation of over $10 billion. According to SpaceX, this funding will be used to support continued innovation in the areas of space transport, reusability, and satellite manufacturing.

CEO Elon Musk announced earlier this month that the company is going to be investing in a satellite internet startup to provide high-speed internet to people on Earth, and also provide the backbone for the internet to eventually reach Mars. That announcement came shortly after Richard Bransons Virgin Group announced a joint investment with Qualcomm into internet satellite company OneWeb.

SpaceX has already made one successful launch this month of its Dragon spacecraft to the International Space Station, delivering supplies, cargo and scientific experiments. Its next launch is scheduled for February, when a Falcon 9 rocket will deliver the Deep Space Climate Observatory, which is geared towards real-time monitoring of solar wind. The monitoring helps develop space weather forecasts to help protect infrastructure points from radiation.

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SpaceX Raises $1 Billion In A Financing Round With Google And Fidelity

MLK Day 2015 Freedom Train arrival at San Francisco Caltrain station, 4th and King – Video


MLK Day 2015 Freedom Train arrival at San Francisco Caltrain station, 4th and King
The Freedom Train is a chartered Caltrain. Freedom Trains were started 30 years ago by Coretta Scott King in honor of her husband. According to news reports, this San Francisco Freedom Train...

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MLK Day 2015 Freedom Train arrival at San Francisco Caltrain station, 4th and King - Video