Research and Markets: Genetics, 6th Edition International Student Version Continues To Educate Today's Students for …

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DUBLIN--(BUSINESS WIRE)--

Dublin - Research and Markets (http://www.researchandmarkets.com/research/z2qzpp/genetics_6th_edit) has announced the addition of John Wiley and Sons Ltd's new book "Genetics, 6th Edition International Student Version" to their offering.

Genetics is one of the most popular texts in use for the introductory course. It opens a window on the rapidly advancing science of genetics by showing exactly how genetics is done. Throughout, the authors incorporate a human emphasis and highlight the role of geneticists to keep students interested and motivated. The sixth edition has been updated to reflect the latest developments in the field of genetics. Genetics continues to educate today's students for tomorrow's science by focusing on features that aid in content comprehension and application.

New to this Edition:

- A new one-column design better showcases important pieces of art and avoids the overwhelmed reaction readers have to the crowded layouts found in many other texts. Boxed sections will be reduced in size to help maintain the flow of the text.

- The Focus On boxes (one per chapter) have been revised to include the most current developments in genetics as well as the most relevant topics to students.

- A streamlined topical coverage, vetted by a panel of Genetics instructors, makes for a text that is manageable in size. Snustad 6e will provide instructors and students with in-depth explanations of only those topics frequently covered in a one-semester course.

- Animated solutions to the solve it problems in the text, utilize Camtasia software. These tutorials provide step-by-step solutions that appear as if they are written-out by hand as an instructor voice-over explains each step.

- Supported by the Wiley Resource kit. The Wiley Resource Kit provides an easy way to provide you with media & assessment. The Resource Kit can be delivered through any Learning Management System (LMS). The offer is free to students, requires no cartridge or CTB license fees from Wiley.

For more information visit http://www.researchandmarkets.com/research/z2qzpp/genetics_6th_edit

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Research and Markets: Genetics, 6th Edition International Student Version Continues To Educate Today's Students for ...

Ontario health system confusing for ailing seniors, study finds

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OTTAWA Ontario's health system is confusing and hard to navigate for the sickest seniors, and a majority have run into problems that lead to poor care, according to a new survey that asks older Ontarians about their health-care experiences.

The report by The Change Foundation, a Toronto think-tank, finds that older voices are often the faintest with many of the seniors surveyed saying they feel ignored or let down by health-care providers, who work in a patchwork of fragmented and badly organized services.

And because of poor information sharing among family doctors, hospitals, nursing homes and home-care agencies, seniors often run into problems, or fall through the cracks entirely, as they are transferred from one provider to another.

The picture that emerges points to shortcomings in a system that remains well suited for patients needing acute, short-term care, but is not equipped to deal with a growing number of seniors suffering from chronic illnesses, who are among the heaviest users of health services.

Instead of reflecting the concerns of health-care insiders, the report gives voice to patients and their families, who are often the best placed to spot problems, said Cathy Fooks, chief executive of The Change Foundation.

"They're really the only people who see the entire trip through the system. Providers interact at various stages in time, but they don't really see the whole process of patients trying to navigate the system."

Of 138 seniors interviewed more than half said they had experienced a disruption in health services because of a communication breakdown among providers, or between providers, themselves and their caregivers.

Only 20 per cent of seniors surveyed said they had never had a problem.

"Seniors and caregivers don't want the moon; they don't necessarily even want more. Their most frequent calls for change revolve around co-ordination and communication," the report said.

The communications gap often starts with family physicians as they refer patients to specialists.

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Ontario health system confusing for ailing seniors, study finds

Walsh-led health bill to be unveiled

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BOSTON Stepping in front of a planned announcement from state senators, House leaders late Thursday said theyll unveil a long-awaited health care payment reform and cost control bill on Friday.

According to House Speaker Robert DeLeos office, the bill will be unveiled during a 2:30 p.m. press conference in Nurses Hall with Health Care Financing Committee Co-chairman Rep. Steven Walsh, D-Lynn, and other members of the committee.

Senate President Therese Murray said last week the Senate planned to debate its version of Gov. Deval Patricks payment reform bill, filed in February 2011, in mid-May, after the bill emerged from the Senate Ways and Means Committee. This week, Murray said the Senate bill would be released next week.

Rumors began circulating on Beacon Hill Wednesday that a House health care bill might emerge on Friday, before the Senate bill. Until Thursday afternoon, House aides either declined to respond or declined comment when asked to confirm those plans or whether committee members were voting on a bill.

After 14 months of review, the Health Care Financing Committee last week released Patricks bill to the Senate, with Walsh saying the panel released the governors bill at the Senates request. Murray on Wednesday expressed surprise at Walshs assertion. On Thursday, committee co-chair Sen. Richard Moore, D-Uxbridge, said he was not aware of any plans by the House to release its own bill.

The Legislature and Gov. Patrick have passed a pair of major health care bills since Gov. Mitt Romney in 2006 signed the landmark law requiring Massachusetts residents to have health insurance or pay tax penalties. While both of those laws included components aimed at addressing rising health care costs, the payment reform bill is viewed as possibly the most aggressive effort to control costs while also striving towards the goal of improving and better coordinating patient care and reducing unnecessary testing.

Many health care industry executives are voluntarily moving toward systems reflecting goals of payment reform financial savings, more integrated and better quality care and a movement away from the fee-for-service payment model and lawmakers must decide whether and how heavily to regulate rates and how much of cost control they should leave to the private sector.

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Hmong health care gap focus of Healthy House dinner

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There's still work to be done when it comes to creating an environment in which Hmong residents are comfortable with Western medicine, including easing cultural and language barriers.

Candice Adam-Medefind, executive director for Healthy House Within a MATCH Coalition in Merced, said "We still have a lot of providers who don't recognize how critical language access is."

To examine what has been done to try to remove those barriers over the last couple of years, the local group will bring in an author who has explored the issue in Merced.

Anne Fadiman wrote "The Spirit Catches You and You Fall Down," which is set in Merced. She will be the keynote speaker at Healthy House's annual multicultural dinner on Saturday at the Merced Elks Club. Four Mercedians featured in the book will participate in a panel discussion with the author.

They will talk about the improvements in the gap between Hmong beliefs and Western medical practices that have been made since the book was published in 1998.

Adam-Medefind said several actions have been taken. "The writing of that book was one of the main events that prompted the setting up of the Healthy House in the community to address the cultural clash between Hmong shamans and the Western medicine establishment," she said.

In addition, the nationally recognized program, Partners in Healing, has made strong contributions to the advancements in the disparities between Hmong beliefs and Western medicine.

The program allows Hmong spiritual healers, known as shamans, to conduct healing ceremonies when requested by Hmong patients or family members to meet their spiritual needs, provide support and promote optimal healing.

The program is made possible by a partnership between the Healthy House and Mercy Medical Center.

In a community as diverse as Merced, culturally competent health care services and language access continue to be an issue, Adam-Medefind said.

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Hmong health care gap focus of Healthy House dinner

Health-care reform panel considers exchange options for Va.

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RICHMOND, Va. --

With the theme from "Mission Impossible" playing in the background, a gubernatorial advisory panel inched closer to deciding what essential health benefits would have to be offered in a Virginia-operated insurance exchange.

The panel, appointed by Gov. Bob McDonnell to help carry out federal health care reform, came just short on Thursday of endorsing a small-group insurance plan offered by Anthem, Virginia's biggest insurer, as the benchmark for competitors in the exchange.

Instead, the Virginia Health Reform Initiative Advisory Council decided to wait for a detailed comparison of benefits currently provided under insurance plans in the state, as well as a better understanding of what services would be covered for mental health, substance abuse and therapies for developmentally delayed children.

"We're almost making it too complicated," said state Sen. John Watkins, R-Powhatan, who has pushed the state to make decisions sooner rather than later on how to establish a health benefits exchange. "It's pretty straight up."

The council debated the benefits issue in its first meeting since making recommendations last September that neither the governor nor the legislature acted upon. McDonnell has not taken a public position on the recommendations, choosing instead to wait until after a U.S. Supreme Court ruling on the Patient Protection and Affordable Care Act.

"Our instructions remain: 'prepare,' " said Health and Human Resources Secretary Bill Hazel, a former orthopedic surgeon who chairs the advisory panel.

Previous recommendations focused on the governing structure for an exchange, including whether to house it at the State Corporation Commission or create an independent, quasigovernmental entity.

The debate on Thursday at the Greater Richmond Convention Center dealt solely with the package of essential benefits that all insurers competing in an exchange would have to offer individuals and small businesses.

Federal regulators have left the decision to the states, while providing a list of 10 essential services that insurers would have to cover, either through a benchmark benefit plan or additional riders that would be eligible for federal subsidies.

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Yes, the Health-Care Mandate Is About Liberty

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Illustration by Ryan Cox

By Jonathan Cohn and David A. Strauss Thu May 03 23:00:36 GMT 2012

As they await the Supreme Court ruling on the Affordable Care Act, legal critics of the law say their case is about liberty. If the government can instruct people to obtain health insurance, they keep asking, whats to stop it from requiring them to buy broccoli?

But the real threat to liberty in this case isnt a hypothetical broccoli law. Its the problem that the mandate remedies -- the failure of the health-insurance market -- and the long-standing national crisis of rising health-care costs that Congress finally found a way to address.

Its not a coincidence that in every advanced country in the world, including the U.S., the government is heavily involved in the health-care market and has been for generations. Everybody needs medical attention, at some point, and virtually everybody needs health insurance to pay for it. Nobody can predict when he or she will need care and virtually nobody can pay for it out of pocket. Even the laws challengers acknowledge these facts.

But in the U.S., not everybody can actually get health insurance -- partly because, as economists have long understood, the health-insurance market is almost uniquely prone to dysfunction.

Insurers need premiums from healthy people, so that, at any one time, they have money to pay the bills of the sick and injured. Private insurers can build these broad risk pools when they sell coverage through large employers, since such companies typically have big and diverse workforces. But when insurers sell health-care policies directly to individuals, they run into trouble: They disproportionately attract people who already have medical conditions.

During the 20th century, this problem of adverse selection pushed many insurers into financial distress.

To preserve themselves, carriers today charge higher premiums, reduce benefits or deny coverage altogether to applicants who have pre-existing medical conditions. Although this keeps insurers solvent, it excludes people who need insurance the most -- in ways that limit their ability to participate fully as members of society and, for that matter, to engage in interstate commerce. Frequently these people cant switch jobs or start a business. In the worst cases, they cant pay their medical bills or obtain the care they need.

By establishing the mandate, which is really just a financial incentive for people to get insurance, the Affordable Care Act will build large, stable risk pools for health insurance. It will also enable the government to set rules about standard benefits and pricing that allow people buying insurance on their own to comparison-shop. In the long run, according to the Congressional Budget Office, it will help government control the cost of medical care, which increasingly strains public and private resources alike -- and today accounts for one-sixth of the American economy.

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Yes, the Health-Care Mandate Is About Liberty

Scuttling health care act will freeze Medicare, White House warns

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WASHINGTON -- Medicare's payment system, the unseen but vital network that handles 100 million monthly claims, could freeze if President Barack Obama's health care law is summarily overturned, the administration quietly informed the courts.

Although Obama's overhaul made significant cuts to providers and improved prescription and preventive benefits, Medicare was overlooked in Supreme Court arguments that focused on the law's controversial requirement that all individuals carry health insurance.

Havoc for Medicare could have repercussions as both parties avidly court seniors in this election year and as hospitals and doctors increasingly complain the program doesn't pay enough.

In papers filed with the Supreme Court, administration lawyers warned of "extraordinary disruption" if Medicare is forced to unwind countless transactions that are based on payment changes required by more than 20 separate sections of the Affordable Care Act.

Opponents say the whole law must go. The administration counters that even if the court strikes down the insurance mandate, it should preserve most of the rest of the legislation. That would leave in place the changes to Medicare and a major expansion of Medicaid coverage.

Last year, in a lower court filing, Justice Department lawyers said reversing Medicare payment changes "would impose staggering administrative burdens" on the government and "could cause major delays and errors" in claims payment.

Former program administrators disagree on the potential for disruptions, while some private industry executives predict an avalanche of litigation unless Congress intervenes.

The AARP says it's concerned. If doctors became embroiled in a legal battle over payments, then "a general concern would be that physicians would cease to take on new Medicare patients, as well as potentially have issues seeing their current patients," said Ariel Gonzalez, an AARP lobbyist.

Medicare payment policies are set through a time-consuming process that begins with legislation passed by Congress. Even if the law were completely overturned, the government would have authority under previous legislation to pay hospitals, doctors, nursing homes and other providers.

But reversing the new law's payment changes from one day to the next would be a huge legal and logistical challenge and raise many questions. How would Medicare treat payments made over the last two years, when the overhaul was the law of the land? Would providers who received cuts subsequently have a right to refunds?

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Scuttling health care act will freeze Medicare, White House warns

Research and Markets: Personalized Medicine: Companies, Trends and World Market

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DUBLIN--(BUSINESS WIRE)--

Dublin - Research and Markets (http://www.researchandmarkets.com/research/vw2h92/personalized_medic) has announced the addition of the "Personalized Medicine: Companies, Trends and World Market" report to their offering.

This broad, high-level report analyzes the expanding Personalized Medicine market. This world market includes important core medical product areas that will continue to have a powerful impact on current and future healthcare delivery. This business report examines key market segments such as targeted drugs and key personalized medicine diagnostics, including companion diagnostic IVDs, LDTs, diagnostic services and related tools or technologies.

Many people already know about DNA, genes and the human genome. The science driving personalized medicine includes pharmacogenetics, pharmacoproteomics and pharmacometabalomix. Personalized medicine uses a targeted drug that depends on the patient information identified by a companion diagnostic (genetic biomarker test). The companion diagnostic identifies which patients would likely benefit from a particular therapy or those who might suffer from a bad side effect. The test information enables doctors to select the drug therapy that would benefit the patient. Drug developers in clinical trials could use a companion diagnostic to select patents that would benefit from a targeted drug.

The report discusses important technologies, including microarray, next-generation sequencing, PCR, bioinformatics, nanotechnology and other platforms. This section highlights key platforms and selected vendors. For example, the field of clinical next generation sequencing is expected to have an impact on personalized medicine.

The report covers subjects including important personalized medicine concepts. The study discusses key biomarkers, commercial diagnostics and therapeutics that drive personalized medicine. The study highlights new personalized diagnostics. This research examines the current targeted therapeutics on the market and drugs in the clinical pipeline.

The report highlights major government regulatory activities that involve personalized medicine in the US and Europe. The US FDA and the European EMA have drafted guidance papers to help drug makers and diagnostic firms develop future targeted therapies guided by companion diagnostics. The recent FDA approvals of Pfizer's Xalkori for lung cancer and Roche's Zelboraf for melanoma demonstrate that a surge in new targeted drugs is happening.

This report is in an interactive PDF format. The interactive feature uses hyperlinks that enable the reader to click the mouse to jump from Table of Contents items to sections inside the report. The hyperlinks also allow the reader to click on links to Internet information.

This study discusses important personalized medicine topics and provides the reader with key findings. The report estimates that the world personalized medicine market value will reach multi-billions of dollars in 2012, with a strong double-digit growth rate. This study reviews the activities of 31 companies.

The report uses 71 figures and tables to help the reader scan the details of major trends, market segmentation, forecast, M&As, partnerships, grant funding, patents and so on. This must-have report would benefit people with job titles including CEO, VPs, Director of Business Development, Research Directors, Entrepreneurs, Venture Capitalists, Investment Planners, Research Scientists, Consultants or Industry Analysts.

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Research and Markets: Personalized Medicine: Companies, Trends and World Market

Naturally blond hair in Solomon Islanders rooted in native gene, Stanford study finds

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Public release date: 3-May-2012 [ | E-mail | Share ]

Contact: Rosanne Spector manishma@stanford.edu 650-725-5374 Stanford University Medical Center

STANFORD, Calif. The common occurrence of blond hair among the dark-skinned indigenous people of the Solomon Islands is due to a homegrown genetic variant distinct from the gene that leads to blond hair in Europeans, according to a new study from the Stanford University School of Medicine.

"This is one of the most beautiful examples to date of the mapping of a simple genetic trait in humans," said David Reich, PhD, a professor of genetics at Harvard University, who was not involved in the study.

The study identifying the gene responsible for blond hair in the Solomon Islands, a nation in the South Pacific, represents a rare case of simple genetics determining human appearance, and shows the importance of including understudied populations in gene mapping studies, said co-senior author Carlos D. Bustamante, PhD, professor of genetics at Stanford. The findings will be published May 4 in Science.

"Since most studies in human genetics only include participants of European descent, we may be getting a very biased view of which genes and mutations influence the traits we investigate. Here, we sought to test whether one of the most striking human traits, blond hair, had the same or different genetic underpinning in different human populations," Bustamante said.

Globally, blond hair is rare, occurring with substantial frequency only in northern Europe and in Oceania, which includes the Solomon Islands and its neighbors. "Its frequency is between 5 and 10 percent across the Solomon Islands, which is about the same as where I'm from," said co-first author Eimear Kenny, PhD, who was born in Ireland.

Many assumed the blond hair of Melanesia was the result of gene flow a trait passed on by European explorers, traders and others who visited in the preceding centuries. The islanders themselves give several possible explanations for its presence, said co-senior author Sean Myles, PhD, a former Stanford postdoctoral scholar who is now an assistant professor at the Nova Scotia Agricultural College. They generally chalked it up to sun exposure, or a diet rich in fish, he said.

After researchers at UCSF generated genetic data from the samples, Kenny, a postdoctoral scholar in Bustamante's lab, began the analysis in September 2010, the week she started at Stanford. "Within a week we had our initial result. It was such a striking signal pointing to a single gene a result you could hang your hat on. That rarely happens in science," she said. "It was one of the best experiences of my career."

In terms of genetic studies, the analysis was straightforward, said Kenny. But gathering the data, accomplished in 2009 by Myles and co-first author Nicholas Timpson, PhD, was more difficult. Much of the Solomon Islands is undeveloped, without roads, electricity or telephones. It's also one of the most linguistically diverse nations in the world, with dozens of languages spoken.

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Naturally blond hair in Solomon Islanders rooted in native gene, Stanford study finds

New genetic line of blond hair discovered

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MICHAEL FIELD/Fairfax NZ

BLOND BEAUTY: A Solomon Island boy displays his locks.

A new genetic line in blond hair has been discovered in an unlikely place - among the people of Melanesia in the Solomon Islands and Fiji.

The magazine Science reports today that scientists now realise that blond hair evolved independently at least twice in human history.

Around 10 per cent of Solomon Islanders had the blond gene, said study author Sean Myles, a geneticist at Nova Scotia Agricultural College in Truro, Canada.

Strikingly there was almost no variation in shades of blond hair.

"It looked pretty obvious to me that it was a real binary trait. You either had blond hair or you didn't," Myles told Science.

After testing 1209 Solomon Islanders scientists compared the entire genetic makeup of 43 blond and 42 dark-haired islanders.

The two groups, they found, had different versions of a crucial gene, TYRP1, one that coded for a protein involved in pigmentation. Switching one "letter" of genetic code - replacing a "C" with a "T" - meant the difference between dark hair and blond hair. A similar mutation creates blond mice by reducing the melanin content in their fur.

The gene was recessive, which meant that blonds inherited it from both parents.

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New genetic line of blond hair discovered

Genetic pathway of rare facial malformation in children pinpointed

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ScienceDaily (May 3, 2012) Researchers at Seattle Children's Research Institute and their collaborators have discovered a pair of defective genes that cause a rare congenital malformation syndrome that can make it impossible for the child to breathe or eat properly without reparative surgery. In a study led by Michael L. Cunningham, MD, PhD, medical director of the Seattle Children's Hospital's Craniofacial Center, a research team pinpointed two genes known as PLCB4 and GNAI3 in a genetic pathway that affects children with auriculocondylar syndrome (ACS). ACS is a rare disorder in which a child's bottom jaw develops as an upper jaw and, in some cases, incorrectly fuses to the base of the skull.

As part of the study, the DNA of five children with similar facial features characteristic of ACS was sequenced. Cunningham and his colleague Mark J. Rieder, PhD (University of Washington) used exome sequencing, selectively sequencing those regions of the patients' DNA believed to constitute the majority of disease-causing mutations. The study, to be published in the May edition of American Journal of Human Genetics, is one of the first genomic studies to identify causative mutations in two genes for the same disorder in the same pathway in a single analysis, Dr. Cunningham said.

While children with ACS have normal cognitive development, severe cases may require an immediate tracheostomy, feeding tubes, and ultimately extensive facial reconstructive surgery to allow them to eat and breathe properly.

"Although ACS is rare, our findings suggest that these genes may also play a role in more common disorders of the jaw and ears," said Dr. Cunningham, who is also chief of the division of craniofacial medicine and professor of pediatrics in the Department of Pediatrics at the University of Washington School of Medicine. "It's possible that more common jaw problems, like the lower jaw abnormality known as Robin sequence and other skull and facial abnormalities such as craniofacial microsomia, are also caused by genes in this pathway."

ACS, a syndrome first described by scientists in 1978, is believed to affect less than one in 50,000 births, though the precise frequency is not known. It is not uncommon for the condition to be misdiagnosed or for diagnosis to be delayed. According to Dr. Cunningham it was the precision of case choice that allowed this discovery.

Of the five cases studied, two of the parents did not have this condition but were carriers for the mutation. "Now that we know the genetic pathway for ACS, we will be able to better identify and counsel people who have normal facial appearances but carry these genes, about the likelihood of passing on this mutation to their children,'' Dr. Cunningham said.

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Genetic pathway of rare facial malformation in children pinpointed

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Autumn-sown sugar beet crops could be included in UK rotations in the future, but only through the development of bolting-resistant varieties using genetic engineering.

A recent British Sugar and Farmers Weekly study tour to Spain visited autumn-sown beet situated in the south of the country, and despite the milder climate being less conducive to bolting, it begs the question: could it work in the UK?

Spain currently grows in excess of 30,000ha of sugar beet, of which about 7,600ha is established in the autumn in the southern region of Andalucia.

"One of the advantages of winter beet in the south is the lower water necessity compared with spring beet, due to the use of winter rains," says Rodrigo Morillo-Velarde, director at AIMCRA.

"Drilling in the autumn period also ensures that the maximum solar radiation coincides with maximum leaf area index, so the crop has its highest production potential.

"We do have problems with randomness of pests and diseases due to the climatic variations, high competition from weeds that germinate throughout the autumn, winter and spring and also some bolting," he says.

Despite bolting in the unlikely event of extremely cold weather, it is something that can be controlled by using conventionally-bred varieties with bolting resistance, adds Mr Morillo-Velarde.

It is the use of the water and sunlight during the months of April, May and June with a large canopy that would appeal to UK growers, which would subsequently increase yield potential.

"It would be such a huge genetic move for the UK, as you are moving the drilling period ahead of the vernalisation conditions during the winter, so realistically, it is something that only GM technology could facilitate," says Patrick Jarvis, best practice agriculture manager with ABSugar.

"What comes with that is all the usual problems associated with genetic engineering, such as government and public opinion. British Sugar would only use the technology if it received the approval from its customers.

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Devangshu Datta: Towards an HIV cure

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Devangshu Datta: Towards an HIV cure Advances in genetic engineering techniques may finally help us win the battle against this global scourge Devangshu Datta / New Delhi May 04, 2012, 00:53 IST

Since AIDS, or acquired immune deficiency syndrome, was identified in 1981, there has been only one medically-certified cure. That occurred under unusual circumstances and it gave researchers an important clue about new ways to attack the disease. Recent advances in genetic engineering techniques have aided in this process. Some studies offer new hope of a cure for the 35 million estimated to be infected worldwide.

No disease inspires as much superstitious dread. So far, AIDS is estimated to have killed over 30 million people and it infects millions every year. It is especially prevalent in Sub-Saharan Africa.

HIV is transmitted through the exchange of body fluids. Common causes of infection (not necessarily in order) include unprotected sex, blood transfusions, sharing needles and so on. The associations with promiscuity and drug addiction make it hard to implement policies to stop HIV-spread. What works best is a combination of sex education and drug awareness programmes, coupled with easy availability of condoms and disposable needles. But in conservative societies like India, people object to sex education. Some religions also discourage the use of condoms.

Someone infected with HIV (HIV-positive) may survive years, without symptoms. The virus attacks a class of white blood cells called CD4 T-cells. It inserts itself into the cell and replicates. T-cells are part of the natural immune system. Once AIDS develops owing to HIV taking over T-cells, the immune system shuts down. Most AIDS patients die of cancer, pneumonia, or some other infection.

The new approaches involve inserting immune genes into HIV-positive patients, through genetic engineering of stem cells. Every researcher is cautious about claims of cures. The characteristic long symptom-less periods and HIVs ability to hide can be cruelly deceptive. HIV-positive people are also vulnerable to quacks. Many charlatans, including a cross-dresser who teaches yoga on Indian television, have claimed at various times to have found AIDS cures.

Some people have natural genetic immunity for various reasons. Advances in understanding of genomes have helped identify some of the causes of immunity. Researchers have known for a while that a mutated gene called CCR5 Delta 32 offers natural immunity to HIV.

The mutation is rare and found only in a few northern Europeans. The normal CCR5 gene, which most people possess, is the receptor HIV uses to enter T-cells. HIV cannot use the Delta-32 mutated gene and, hence, cannot replicate in a host who has two copies of the CCR5 Delta 32 gene (one inherited from each parent). Even one copy of Delta 32 seems to offer some protection. Only about one per cent of northern Europeans possess both copies.

In 2007, Timothy Ray Brown, an American resident in Berlin, was HIV-positive and also under treatment for leukaemia. Leukaemia causes an abnormal increase in white blood cells and a drop in red cells. Blood cells are produced by bone marrow. One drastic treatment is a bone marrow stem cell transplant from a healthy person. This helps regenerate healthy blood with a good haemoglobin ratio, and a new immune system. Its dangerous since the patients entire immune system must be destroyed prior to the transplant.

Browns doctors at the Charite University Medicine Berlin, Kristina Allers and Gero Hutter, found a compatible donor who belonged to that rare one per cent with the Delta-32 mutation. Five years later, after the transplant procedures, the Berlin Patient, as Brown is called in medical journals, is still HIV-free and doctors concur that this is a functional cure.

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Devangshu Datta: Towards an HIV cure

AIDS gene therapy safe — is it a "cure"?

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By Robert Bazell, Chief science and medical correspondent, NBC News

One step closer to a cure for AIDS that is the implication of results out Wednesday from from several leading research centers.

It should be noted that many people involved in AIDS research, including several who carried out the latest research, avoid the c word. Their goal is to allow people infected with HIV to live without daily doses of the medications that usually keep the virus under control-- at a large financial cost --and a risk of side effects.

The latest work, published in Science Translational Medicine, details 43 HIV-infected volunteers who had experimental genes inserted into their disease-fighting white blood cells 11 years ago. All patients are doing fine. After more than a decade with this gene therapy, there are no side effects. In almost every one the inserted genes are still working properly.

While these experiments were never intended to treat or cure anything, they lay the groundwork for gene therapy that could have a substantial impact on HIV disease.

A cure for AIDS became an obvious goal as soon as the disease was discovered 30 years ago. But it became the dirty four letter word, as Jon Cohen of Science magazine put it, after some spectacular failures. Soon after the powerful cocktail of anti-AIDS drugs came on the market in 1996, some scientists speculated they could use the drugs to knock out all the infection in the body. But that idea crashed as repeated experiments showed that pockets of infected cells hid in various parts of the body, emerging quickly as soon as the drugs were withdrawn.

HIV has infected some 50 million people in the world and none has been cured -- except perhaps Timothy Ray Brown.

It was the case of Brown, also known as the Berlin patient, that energized the new search for a cure.Infected with HIV, Brown was dying not of that disease, but of leukemia. His only hope was a bone marrow transplant first killing, then replacing all the cells in his body that make blood cells with those from a donor. Browns doctor Gero Htter was not an AIDS specialist, but he knew that about 1 percent of people of European decent have a mutation in a receptor called CCR5 on certain white blood cells that make HIV infection very difficult.So the doctor sought a donor with that mutation.

The transplant took place in 2007. In 2010 Htter published his results. Not only had the transplant eliminated Browns leukemia, he no longer needed to take his HIV medications and the most sophisticated tests find no trace of HIV in his body.

A transplant with a serious risk of death, costing more than $250,000, will not be a treatment for a disease contained by medications.But the case raised the possibility that modifying the white blood cells with gene therapy might do the trick. Several experiments are underway in both animals and humans and more are planned.

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AIDS gene therapy safe -- is it a "cure"?

Families of SMA Awards New Funding to Advance a CNS Delivered Gene Therapy for Spinal Muscular Atrophy

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ELK GROVE VILLAGE, Ill., May 2, 2012 (GLOBE NEWSWIRE) -- Families of SMA is pleased to announce the award of up to $750,000 for an important new grant to Dr. Brian Kaspar at Nationwide Children's Hospital. This award will support preclinical development of a CNS-delivered Gene Therapy for Spinal Muscular Atrophy. With funding from FSMA, Dr. Kaspar's team will initiate the studies needed for an Investigational New Drug (IND) application for this therapy to the Food and Drug Administration (FDA).

"Families of SMA is excited to be awarding new goal-directed drug discovery funding for this gene therapy program. This work follows up on a 2010 grant from FSMA to test the age-dependence in primates of this gene therapy. The new funding will allow us to accomplish several key goals simultaneously", says Jill Jarecki, PhD, FSMA Research Director. "First, it will allow us to advance this very promising new therapy for SMA towards human clinical trials. Second, it will allow FSMA to fund multiple SMA drug programs concurrently, which have different approaches. Doing this will increase our community's chances of successfully finding a treatment for SMA."

"This is extremely important funding from FSMA to allow us to collect additional pre-clinical data for a CNS delivered AAV gene delivery into the cerebrospinal fluid, which will be important information to present to the FDA. It also jump starts research prior to obtaining government and commercial involvement which we are actively pursuing. We are quite hopeful for a positive funding decision on a recent NIH proposal for co-funding of this project with FSMA." Brian Kaspar, PhD, Associate Professor, Principal Investigator The Research Institute at Nationwide Children's Hospital, The Ohio State University.

The overall project goals are: 1) to optimize the dosing regimen for CNS-delivered SMA gene therapy; 2) to conduct the GLP toxicology, immune response, and bio-distribution experiments required by the FDA; 3) to prepare and hold a pre-IND meeting with the FDA; 4) to submit an IND to the FDA to begin human clinical trials; and 5) to produce clinical grade material for human studies. The overall timeline for this work is expected to be three years.

This Program was chosen for funding by the FSMA Translational Advisory Committee (TAC), after reviewing multiple potential new drug programs. Every drug program carries risk of encountering hurdles at each of the stages described above. Therefore, a project specific Steering Committee has been put in place, which is comprised of experts in both gene therapy and in SMA biology, with representatives from academia and industry.

This committee will help manage the project, ensuring it progresses in an efficient and well-run manner. In addition, project funding will be awarded upon meeting predetermined milestones, decided on by the Steering Committee.

"I am incredibly excited by FSMA's decision to support Dr. Kaspar and his team in this very important project. As a pharmaceutical scientist who works every day in drug discovery and development, I am encouraged by the quality of the science and the fact that it aims to address SMA treatment from a different vantage point from other programs in the SMA drug pipeline. This is only the first step, but it's a critically important step toward assessing whether gene therapy is a viable approach in SMA. Time will tell but I, for one, am incredibly hopeful and look forward to working with FSMA to facilitate the efforts of Dr. Kaspar and his team. I should add that as a parent of an SMA child, I am always looking for a medical breakthrough that could the transform the lives of SMA patients." Timothy P. Reilly, PhD, DABT Director, Drug Safety Evaluation, Bristol-Myers Squibb. TAC Member.

A major goal at FSMA has been to build the SMA drug pipeline, and we have been investing in drug research since 2000 towards this goal. Even with our community's current progress in adding programs to the SMA drug pipeline and advancing programs to start clinical trials, FSMA believes it is critical to do more. Statistics show that only 10% of all drugs initiating human clinical trials ultimately receive FDA approval. The new funding announced here by FSMA for this preclinical drug program will help achieve this goal. FSMA has been involved in funding half of all the ongoing novel drug programs for SMA.

About Families of Spinal Muscular Atrophy

Families of SMA funds and directs the leading SMA research programs to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $50 million in research and been involved in funding half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 85,000 members and supporters throughout the United States, and is dedicated to creating a treatment and cure by: funding and advancing a comprehensive research program; supporting SMA families through networking, information and services; improving care for all SMA patients; educating healthcare professionals and the public about SMA; enlisting government support for SMA; embracing all touched by SMA in a caring community. For more information: http://www.curesma.org.

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Families of SMA Awards New Funding to Advance a CNS Delivered Gene Therapy for Spinal Muscular Atrophy

A Media Event on Clinical Developments in Gene and Cell Therapy

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Academic and industry leaders in gene and cell therapy will be featured at a Media Event in Philadelphia, PA on May 15, 2012, immediately preceding the 15th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) on May 16-19, 2012.Milwaukee, WI (PRWEB) May 02, 2012 Academic and industry leaders in gene and cell therapy will be featured at a Media Event in Philadelphia, PA on May ...

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A Media Event on Clinical Developments in Gene and Cell Therapy

A Step Forward For Gene Therapy To Treat HIV

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PLoS Biology

HIV particles assemble at the surface of a white blood cell called a macrophage.

Millions of people around the world are living with HIV, thanks to drug regimens that suppress the virus. Now there's a new push to eliminate HIV from patients' bodies altogether. That would be a true cure.

We're not there yet. But a report in Science Translational Medicine is an encouraging signpost that scientists may be headed in the right direction.

Forty-three patients got immune cells designed to attack and kill cells infected with HIV. As long as 16 years later, these genetically engineered T cells are still circulating in their bloodstreams. And there's been no sign the gene therapy caused any cancers, or is likely to.

That may seem like a modest victory. After all, there's no evidence yet that the gene therapy did what it's supposed to eliminate the reservoir of HIV hiding in the patients' cells, waiting to emerge as soon as patients stop taking their antiviral drugs.

But to scientists in HIV and gene therapy research, it's a highly encouraging indicator. "We're not hitting a home run. This is a single," AIDS researcher Pablo Tebas of the University of Pennsylvania tells Shots.

"It looks like if you do this, it's going to be safe because we have not seen any toxicity in 16 years," he says. "And two, the genetically modified cells are still circulating. They perpetuate. Those are two important things this study is telling us."

Tebas is not an author of the study, but he works with the Penn researchers who did the work. They were unavailable for comment.

Previous attempts at this kind of gene therapy, called adoptive T cell transfer, have been plagued by cancers that can arise when the genes introduced into engineered cells insert themselves next to growth-promoting genes. In other cases the engineered cells have died out before they have a sustained positive effect.

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A Step Forward For Gene Therapy To Treat HIV