Monthly Archives: February 2014

Mexican fisherman shore up Marshall Islands castaway's account

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A man lost at sea for more than a year survived, he says, on only what he caught from a small fishing boat. It seems improbable, but doctors say it's possible.

By Tracy Connor, Staff Writer, NBC News

The father of Pacific castaway Jose Salvador Alvarenga said he was told his long-lost son vanished on a fishing trip but he didnt have the heart to break the news to his ailing wife.

Courtesy of Telemundo

Ezequiel Cordoba saw Jose Salvador Alvarenga on Dec. 18, 2012, when he left to go fishing with Jose Salvador Alvarenga.

Now both parents are celebrating after learning that the son they last saw almost a decade ago is alive after washing up on the Marshall Islands with a long beard and an astonishing story of more than a year lost at sea.

They told me that he had entered the sea and that hed never come out, Ricardo Orellana told NBC partner Telemundo from the family home in El Salvador.

But because she was ill, I told her nothing, he said of his wife, Julia Alvarenga, who wept tears of gratitude.

Although she had no idea that Alvarenga had left Mexico on a 24-foot boat and never returned, because he had been out of touch for so long, she worried misfortune had befallen him.

I pleaded to my all powerful God that if my son was still alive, that he would take care of him. If he was dead, that he would forgive him, she said.

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Mexican fisherman shore up Marshall Islands castaway's account

Human Genetics – Buzzle

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Human genetics is the branch that studies the aspect of 'inheritance' in formation of human beings. Its study makes it easier to understand the cause of certain disorders, behavioral issues and development.

The Importance of Housekeeping Genes

Housekeeping genes are those that are always expressed in all cells of an organism, and are important for the maintenance of basic or 'housekeeping' functions of the cell. This Buzzle write-up provides a brief account of the same.

Pros and Cons of Gene Patenting

Gene patenting is quite common in the medical field today, however, it is still surrounded by many controversies. In this Buzzle article, we will discuss what is gene patenting and what are its positive and negative aspects.

Pros and Cons of Designer Babies

Designer babies are babies, whose genetic makeup has been artificially screened and chosen by scientists, via genetic engineering. This concept has raised numerous ethical issues. Let's have a look at the pros and cons of designer...

DNA Replication Steps

The process of DNA replication comprises a set of carefully orchestrated sequence of events to duplicate the entire genetic content of a cell. The current article provides a short insight into the complex DNA replication steps.

Incomplete Dominance Examples

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Human Genetics - Buzzle

Removing Hair Around the Base of a Cat’s Tail : Cat Health Care & Behavior – Video

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Removing Hair Around the Base of a Cat #39;s Tail : Cat Health Care Behavior
Subscribe Now: http://www.youtube.com/subscription_center?add_user=Expertvillage Watch More: http://www.youtube.com/Expertvillage Removing hair around the ba...

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Removing Hair Around the Base of a Cat's Tail : Cat Health Care & Behavior - Video

Obamacare Success Story: LA Jewish organization serving seniors salutes heathcare reforms – Video

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Obamacare Success Story: LA Jewish organization serving seniors salutes heathcare reforms
The Affordable Care Act, also known as Obamacare, has been quite controversial especially when it comes to getting Americans registered online. But there hav...

By: JewishNewsOne

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Obamacare Success Story: LA Jewish organization serving seniors salutes heathcare reforms - Video

Health care navigator available locally

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Signing up for affordable health care will be a little bit easier through the end of March because of a partnership between Washington-Morgan Community Action and the Ohio Association of Foodbanks.

The two organizations teamed up in October to offer a free health care enrollment process through a navigator grant.

According to Carrie McNamee, director of senior and community services for Washington-Morgan Community Action, 36 people have been helped by the health care navigator.

"We definitely want to encourage people to call if they have a question," she said, adding that the hope was to help many more people before the March 31 deadline to sign up.

The navigator can answer questions over the phone or local residents can make an appointment and go to Community Action to meet in person and review the website with a helping hand.

Joree Jacobs, director of communications for the Ohio Association of Foodbanks, said there is a need for affordable health care and the assistance provided is just an extension of the services the organization offers.

Assistance is provided by Washington-Morgan Community Action and the Ohio Association of Foodbanks.

For local assistance in answering questions or setting up an appointment, call Community Action at 373-3745.

Navigators will help those seeking assistance navigate the federal website and answer the questions, but it is up to each individual to decide which plan is right for them.

Each individual will need to know budgetary and tax information going into the appointment.

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Health care navigator available locally

Health care law may lead some to quit jobs, report says

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WASHINGTON Several million American workers will cut back their hours on the job or leave the nation's workforce entirely because of President Barack Obama's health care overhaul, congressional analysts said Tuesday, adding fresh fuel to the political fight over "Obamacare."

The workforce changes would mean nationwide losses equal to 2.3 million full-time jobs by 2021, in large part because people would opt to keep their income low to stay eligible for federal health care subsidies or Medicaid, the Congressional Budget Office said. It had estimated previously that the law would lead to 800,000 fewer jobs by that year.

Republican lawmakers seized on the report as major new evidence of what they consider the failures of Obama's overhaul, the huge change in U.S. health coverage that they're trying to overturn and planning to use as a main argument against Democrats in November's midterm elections.

It's the latest indication that "the president's health care law is destroying full-time jobs," said Republican Rep. John Kline of Minnesota, chairman of the House Education and the Workforce Committee. "This fatally flawed health care scheme is wreaking havoc on working families nationwide."

But the White House said the possible reduction would be due to voluntary steps by workers rather than businesses cutting jobs people having the freedom to retire early or spend more time as stay-at-home parents because they no longer had to depend only on their employers for health insurance.

The law means people "will be empowered to make choices about their own lives and livelihoods," said White House Press Secretary Jay Carney.

CBO Director Douglas Elmendorf said the top reasons people would reduce work would be to qualify for subsidized coverage and an expanded Medicaid program. But he said lower wages because of penalties on employers who don't provide coverage and looming taxes on generous health care plans would also be a factor.

The agency also reduced its estimate of the number of uninsured people who will get coverage through the health care law. The budget experts now say 1 million more people will be uninsured this year than had been expected, partly because of the website problems that prevented people from signing up last fall.

However, it wasn't all bad news for the Obama administration. The CBO's wide-ranging report predicted that the federal budget deficit will fall to $514 billion this year, down from last year's $680 billion and the lowest by far since Obama took office five years ago.

The new estimates also say the health care law will, in the short run, benefit the economy by boosting demand for goods and services because the lower-income people it helps will have more purchasing power. The report noted that the 2014 premiums that people pay for exchange coverage are coming in about 15 percent lower than projected, and the health care law, on balance, still is expected to reduce the federal deficit.

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Health care law may lead some to quit jobs, report says

Health care law will mean fewer people on the job – NBC40.net

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By ANDREW TAYLOR and RICARDO ALONSO-ZALDIVAR Associated Press

WASHINGTON (AP) - Several million American workers will cut back their hours on the job or leave the nation's workforce entirely because of President Barack Obama's health care overhaul, congressional analysts said Tuesday, adding fresh fuel to the political fight over "Obamacare."

The workforce changes would mean nationwide losses equal to 2.3 million full-time jobs by 2021, in large part because people would opt to keep their income low to stay eligible for federal health care subsidies or Medicaid, the Congressional Budget Office said. It had estimated previously that the law would lead to 800,000 fewer jobs by that year.

Republican lawmakers seized on the report as major new evidence of what they consider the failures of Obama's overhaul, the huge change in U.S. health coverage that they're trying to overturn and planning to use as a main argument against Democrats in November's midterm elections.

It's the latest indication that "the president's health care law is destroying full-time jobs," said Republican Rep. John Kline of Minnesota, chairman of the House Education and the Workforce Committee. "This fatally flawed health care scheme is wreaking havoc on working families nationwide," he said.

But the White House said the possible reduction would be due to voluntary steps by workers rather than businesses cutting jobs - people having the freedom to retire early or spend more time as stay-at-home parents because they no longer had to depend only on their employers for health insurance.

The law means people "will be empowered to make choices about their own lives and livelihoods," said White House Press Secretary Jay Carney.

CBO Director Douglas Elmendorf said the top reasons people would reduce work would be to qualify for subsidized coverage and an expanded Medicaid program but that lower wages - because of penalties on employers who don't provide coverage and looming taxes on generous health care plans - would also be a factor.

The agency also reduced its estimate of the number of uninsured people who will get coverage through the health care law. The budget experts now say 1 million more people will be uninsured this year than had been expected, partly because of the website problems that prevented people from signing up last fall.

However, it wasn't all bad news for the Obama administration. The CBO's wide-ranging report predicted that the federal budget deficit will fall to $514 billion this year, down from last year's $680 billion and the lowest by far since Obama took office five years ago.

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Health care law will mean fewer people on the job - NBC40.net

Health care law to reduce labor participation – NBC40.net

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By ANDREW TAYLOR and RICARDO ALONSO-ZALDIVAR Associated Press

WASHINGTON (AP) - Several million American workers will cut back their hours on the job or leave the nation's workforce entirely because of President Barack Obama's health care overhaul, congressional analysts said Tuesday, adding fresh fuel to the political fight over "Obamacare."

The workforce changes would mean nationwide losses equal to 2.3 million full-time jobs by 2021, in large part because people would opt to keep their income low to stay eligible for federal health care subsidies or Medicaid, the Congressional Budget Office said. It had estimated previously that the law would lead to 800,000 fewer jobs by that year.

Republican lawmakers seized on the report as major new evidence of what they consider the failures of Obama's overhaul, the huge change in U.S. health coverage that they're trying to overturn and planning to use as a main argument against Democrats in November's midterm elections.

It's the latest indication that "the president's health care law is destroying full-time jobs," said Republican Rep. John Kline of Minnesota, chairman of the House Education and the Workforce Committee. "This fatally flawed health care scheme is wreaking havoc on working families nationwide," he said.

But the White House said the possible reduction would be due to voluntary steps by workers rather than businesses cutting jobs - people having the freedom to retire early or spend more time as stay-at-home parents because they no longer had to depend only on their employers for health insurance.

The law means people "will be empowered to make choices about their own lives and livelihoods," said White House Press Secretary Jay Carney.

CBO Director Douglas Elmendorf said the top reasons people would reduce work would be to qualify for subsidized coverage and an expanded Medicaid program but that lower wages - because of penalties on employers who don't provide coverage and looming taxes on generous health care plans - would also be a factor.

The agency also reduced its estimate of the number of uninsured people who will get coverage through the health care law. The budget experts now say 1 million more people will be uninsured this year than had been expected, partly because of the website problems that prevented people from signing up last fall.

However, it wasn't all bad news for the Obama administration. The CBO's wide-ranging report predicted that the federal budget deficit will fall to $514 billion this year, down from last year's $680 billion and the lowest by far since Obama took office five years ago.

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Health care law to reduce labor participation - NBC40.net

Penn study reveals genetics impact risk of early menopause among some female smokers

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PUBLIC RELEASE DATE:

5-Feb-2014

Contact: Katie Delach katie.delach@uphs.upenn.edu 215-349-5964 University of Pennsylvania School of Medicine

PHILADELPHA - New research is lighting up yet another reason for women to quit smoking. In a study published online in the journal Menopause, researchers from the Perelman School of Medicine at the University of Pennsylvania report the first evidence showing that smoking causes earlier signs of menopause in the case of heavy smokers, up to nine years earlier than average in white women with certain genetic variations.

Though previous studies have shown that smoking hastens menopause by approximately one to two years regardless of race or genetic background, this study is the first of its kind to demonstrate that genetic background is significantly associated with a further increased risk of menopause in some white women who smoke. No statistically significant relationships between smoking, the gene variants under investigation and earlier menopause were observed in African American women.

While symptoms of menopause such as hot flashes, anxiety and insomnia can result in discomfort, embarrassment, and irritability, the onset of menopause is also associated with risks of coronary artery disease, osteoporosis, and death from all causes. On average, women enter menopause at around 50 years of age. However, the research team now reports that menopause may begin at an earlier age in white female smokers who are carriers of two different gene variants. While the genes themselves do not result in early onset menopause, variations of the genes CYP3A4*1B and CYP1B1*3 were found to increase the risk of entering menopause at an earlier age in white smokers. The genetic variants were present in seven and 62 percent of white women in the study population, respectively.

"This study could shed new light on how we think about the reproductive risks of smoking in women. We already know that smoking causes early menopause in women of all races, but these new results show that if you are a white smoker with these specific genetic variants, your risk of entering menopause at any given time increases dramatically," said the study's lead author Samantha F. Butts, MD, MSCE, assistant professor of Obstetrics and Gynecology at Penn Medicine.

Results of the study, which enrolled over 400 women aged 35 to 47 from the Penn Ovarian Aging Study, found that in carriers of the CYP3A4*1B variation, the average time-to-menopause after entering the study in heavy smokers, light smokers, and nonsmokers was 5.09 years, 11.36 years, and 13.91 years, respectively. This means that for heavily smoking white females with this genetic background, the average time-to-menopause was approximately nine years earlier than in nonsmoking carriers.

In white carriers of the CYP1B1*3 variation, the average time-to-menopause in heavy smokers, light smokers, and nonsmokers was 10.41 years, 10.42 years, and 11.08 years, respectively -- a statistically significant difference although not as stark as the findings for the CYP3A4*1B variant.

The Penn study did not examine why no statistically significant relationships between smoking, the gene variants under investigation, and earlier menopause were observed in African Americans.

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Penn study reveals genetics impact risk of early menopause among some female smokers

National Poll Shows Public Divided on Genetic Testing to Predict Cancer Risk

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Newswise Salt Lake City, UT A national poll from the University of Utahs Huntsman Cancer Institute shows 34 percent of respondents would not seek genetic testing to predict their likelihood of developing a hereditary cancer even if the cost of the testing was not an issue. Concerns about employment and insurability were cited as the primary reason, even though current laws prohibit such discrimination.

The poll also shows only 35 percent of respondents would be extremely or very likely to seek aggressive prophylactic or preventive treatment, such as a mastectomy, if they had a family history of cancer and genetic testing indicated a genetic pre-disposition to cancer.

I see patients every week who could have taken steps to reduce their risk if theyd known theyd had a predisposition for a certain type of cancer. The best treatment for cancer is prevention, of which genetic testing plays an integral role, said Saundra Buys, M.D., co-director of the Family Cancer Assessment Clinic and medical director of the High Risk Cancer Research at Huntsman Cancer Institute (HCI), and professor of medicine at the University of Utah. In addition to educating the public about the important role genetic testing plays in both prevention and treatment of cancer, we must also work to eliminate perceived false barriers to testing, such as concerns about insurability and employment.

Nearly 40 percent of those who said they wouldnt seek testing reported being somewhat or extremely concerned that the results would impact opportunities for employment, while 69 percent of that same group reported being somewhat or extremely concerned that the results would have an adverse impact on their ability to get insurance.

Inherited mutations play a major role in the development of approximately 5 percent of all cancers. Genetic mutations associated with more than 50 hereditary cancer syndromes including those discovered at the University of Utah for melanoma, colon and breast cancer have been identified.

Buys says the survey demonstrates that even with increased media attention to genetic testing in recent months more work is needed to educate the public about the type of information genetic testing provides and who should seek it. She says family and personal health history are the most important factors in determining whether a person should consider genetic testing.

She warns, however, that genetic testing is only as good as the genetic counseling that accompanies it. There are many genetic tests being ordered in physician offices around the country without the benefit of genetic counseling. The results of these tests are complex, and without appropriate counseling, can cause confusion and unneeded anxiety for patients, said Buys.

Other findings from the poll:

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National Poll Shows Public Divided on Genetic Testing to Predict Cancer Risk

Study Reveals Genetics Impact Risk of Early Menopause Among Some Female Smokers

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Contact Information

Available for logged-in reporters only

Newswise PHILADELPHA - New research is lighting up yet another reason for women to quit smoking. In a study published online in the journal Menopause, researchers from the Perelman School of Medicine at the University of Pennsylvania report the first evidence showing that smoking causes earlier signs of menopause in the case of heavy smokers, up to nine years earlier than average in white women with certain genetic variations.

Though previous studies have shown that smoking hastens menopause by approximately one to two years regardless of race or genetic background, this study is the first of its kind to demonstrate that genetic background is significantly associated with a further increased risk of menopause in some white women who smoke. No statistically significant relationships between smoking, the gene variants under investigation and earlier menopause were observed in African American women.

While symptoms of menopause such as hot flashes, anxiety and insomnia can result in discomfort, embarrassment, and irritability, the onset of menopause is also associated with risks of coronary artery disease, osteoporosis, and death from all causes. On average, women enter menopause at around 50 years of age. However, the research team now reports that menopause may begin at an earlier age in white female smokers who are carriers of two different gene variants. While the genes themselves do not result in early onset menopause, variations of the genes CYP3A4*1B and CYP1B1*3 were found to increase the risk of entering menopause at an earlier age in white smokers. The genetic variants were present in seven and 62 percent of white women in the study population, respectively.

This study could shed new light on how we think about the reproductive risks of smoking in women. We already know that smoking causes early menopause in women of all races, but these new results show that if you are a white smoker with these specific genetic variants, your risk of entering menopause at any given time increases dramatically, said the studys lead author Samantha F. Butts, MD, MSCE, assistant professor of Obstetrics and Gynecology at Penn Medicine.

Results of the study, which enrolled over 400 women aged 35 to 47 from the Penn Ovarian Aging Study, found that in carriers of the CYP3A4*1B variation, the average time-to-menopause after entering the study in heavy smokers, light smokers, and nonsmokers was 5.09 years, 11.36 years, and 13.91 years, respectively. This means that for heavily smoking white females with this genetic background, the average time-to-menopause was approximately nine years earlier than in nonsmoking carriers.

In white carriers of the CYP1B1*3 variation, the average time-to-menopause in heavy smokers, light smokers, and nonsmokers was 10.41 years, 10.42 years, and 11.08 years, respectively -- a statistically significant difference although not as stark as the findings for the CYP3A4*1B variant.

The Penn study did not examine why no statistically significant relationships between smoking, the gene variants under investigation, and earlier menopause were observed in African Americans.

It is possible that uniform relationships among white and African American women were not found due to other factors associated with race that modify the interaction between smoking and genes, said Butts. It is well known that race affects multiple features of menopause, and this could be another. Further investigation is needed to clarify this question.

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Study Reveals Genetics Impact Risk of Early Menopause Among Some Female Smokers

Can Genetic Engineering Mitigate California's Drought?

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STANFORD, CA Water is in increasingly short supply in many parts of the United States. Here in California, where most of the state is experiencing extreme drought, 2013 was the driest year on record, and we have had no relief during what should be the height of the rainy season. Moreover, theres no end in sight: The Climate Prediction Center of the National Weather Serviceforecaststhat the drought will persist or intensify at least through April.

Reservoir levels are dropping, the snow pack is almost nonexistent, and many communities have already imposed restrictions on water usage. In the city of Santa Cruz, for example, restaurants can no longer serve drinking water unless diners specifically request it; Marin County residents have been asked not to clean their cars or to do so only at eco-friendly car washes; and there are limitations on watering lawns in towns in Mendocino County.

But it is the states premier industry farming that will feel the pinch most. In an average year, farmers use 80 percent of the water used by people and businesses, according to the Department of Water Resources.

During a January 19 press conference at which he declared a water emergency, Governor Jerry Brown said of the drought, This is not a partisan adversary. This is Mother Nature. We have to get on natures side and not abuse the resources that we have.

Drought may not be partisan, but it does raise critical issues of governance, public policyand how best to use the states natural resources. It also offers an example of the Law of Unintended Consequences: Ironically, Santa Cruz, Mendocino and Marin counties all of which boast politically correct, far-left politics are among the local jurisdictions that have banned a key technology that could conserve huge amounts of water.

The technology is genetic engineering performed with modern molecular techniques, sometimes referred to as genetic modification (GM) or gene-splicing, which enables plant breeders to make old crop plants do spectacular new things, including conserve water. In the United States and about 30 other countries, farmers are using genetically engineered crop varieties to produce higher yields, with lower inputs and reduced impact on the environment.

Even with R&D being hampered by resistance from activists and discouraged by governmental over-regulation, genetically engineered crop varieties are slowly but surely trickling out of the development pipeline in many parts of the world. Cumulatively, over 3.7 billion acres of them have been cultivated by more than 17 million farmers in 30 countries during the past 15 years without disrupting a single ecosystem or causing so much as a tummy ache in a consumer.

Most of these new varieties are designed to be resistant to herbicides, so that farmers can adopt more environment-friendly no-till farming practices and more benign herbicides; or to be resistant to pests and diseases that ravage crops. Others possess improved nutritional quality. But the greatest boon of all both to food security and to the environment in the long term will likely be the ability of new crop varieties to tolerate periods of drought and other water-related stresses. Where water is unavailable for irrigation, the development of crop varieties able to grow under conditions of low moisture or temporary drought could both boost yields and lengthen the time that farmland is productive.

Even where irrigation is feasible, plants that use water more efficiently are needed. Because irrigation for agriculture accounts for roughly 70 percent of the worlds fresh water consumption, the introduction of plants that grow with less water would allow much of it to be freed up for other uses. Especially during drought conditions, even a small percentage reduction in the use of water for irrigation could result in huge benefits.

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Can Genetic Engineering Mitigate California's Drought?

Gene therapy may be possible cure for Hurler syndrome: Mouse Study

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Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome -- an often fatal condition that causes organ damage and other medical complications.

Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and Stroke (NINDS) report their unique strategy for treating the disease the week of Feb. 3-7 in Proceedings of the National Academy of Sciences (PNAS).

Researchers were able to genetically insert into the cells a gene that produces a critical lysosomal enzyme (called IDUA) and then inject the engineered cells into mice to treat the disorder. Follow up tests showed the treatment resulted in a complete metabolic correction of the disease, according to the authors.

"Our findings demonstrate a unique and somewhat surprising delivery pathway for lysosomal enzymes," said Dao Pan, PhD, corresponding author and researcher in the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's. "We show proof of concept that platelets and megakaryocytes are capable of generating and storing fully functional lysosomal enzymes, which can lead to their targeted and efficient delivery to vital tissues where they are needed."

The mice tested in the study modeled human Hurler syndrome, a subset of disease known as mucopolysaccharidosis type I (MPS I), one of the most common types of lysosomal storage diseases. MPS I is a lysosomal storage disease in which people do not make an enzyme called lysosomal alpha-L-iduronidase (IDUA).

IDUA helps break down sugar molecules found throughout the body, often in mucus and fluids around joints, according to the National Library of Medicine/National Institutes of Health. Without IDUA, sugar molecules build up and cause organ damage. Depending on severity, the syndrome can also cause deafness, abnormal bone growth, heart valve problems, joint disease, intellectual disabilities and death.

Enzyme replacement therapy can be used to treat the disease, but it is only temporary and not curative. Bone marrow transplant using hematopoietic stem cells also has been tested on some patients with mixed results. The transplant procedure can carry severe risks and does not always work.

Pan and her colleagues -- including Roscoe O. Brady, MD, a researcher at NINDS -- report that using platelets and megakaryocytes for gene therapy is effective and could reduce the risk of activating cancer-causing oncogenes in hematopoietic stem cells.

The authors said tests showed that human megakaryocytic cells were capable of overexpressing IDUA, revealing their capacity for potential therapeutic benefit. While engineering megakaryocytes and platelets for infusion into their mouse models of Hurler, the scientists report they were able to release IDUA directly into amply sized extracellular spaces or inside micro-particles as the cells matured or activated. The cells were able to produce and package large amounts of functional IDUA and retained the capacity to cross-correct patient cells.

After infusing mouse models of Hurler with the genetically modified cells, researchers said this led to long-term normalization of IDUA levels in the animal's blood with versatile delivery routes and on-target preferential distribution to the liver and spleen. The treatment led to a complete metabolic correction of MPS I in most peripheral organs of the mice.

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Gene therapy may be possible cure for Hurler syndrome: Mouse Study

Gene therapy pioneer uniQure raises $82 million in U.S. IPO

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(Reuters) - A small Dutch company behind the Western world's first approved gene therapy priced its shares above the expected range in a U.S. stock offering on Wednesday, showing the current investor appetite for biotechnology.

Amsterdam-based uniQure said it would sell 5.4 million shares at an initial public offering price of $17.00 each, netting it $81.9 million after expenses. It had previously indicated a price range of $13.00 to $15.00.

The stock was trading just below the issue price at $16.80 by 1600 GMT.

UniQure won approval in November 2012 to sell its drug Glybera in Europe and intends to start selling it as a treatment for the ultra-rare disease lipoprotein lipase deficiency (LPLD) with partner Chiesi in the first half of 2014.

The drug is likely to break new ground as the world's most expensive medicine, with a potential price tag of more than $1 million. A high price is needed because a single dose could last a lifetime, giving uniQure just one shot at recouping its investment.

Chief Executive Jorn Aldag said in 2012 that Glybera could sell for around 250,000 euros a year for five years, implying a total price of 1.25 million euros ($1.6 million).

The company has since said that no decision has been taken on price and its IPO prospectus also said it now believed that a one-time price, rather than an annuity-based system, was the most likely pricing model.

Glybera is a modified virus that delivers the correct version of a gene into people afflicted with LPLD, a hereditary disorder that raises the risk of potentially lethal inflammation of the pancreas.

Rare or so-called orphan diseases are winning increased attention from drug developers and several products from companies including Sanofi, Shire and Alexion already cost hundreds of thousands of dollars a year.

UniQure is not the first gene therapy firm to float on Nasdaq. Bluebird Bio made its debut last June but the Cambridge, Mass.-based company has yet to win a regulatory green light for its products.

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Gene therapy pioneer uniQure raises $82 million in U.S. IPO

Mouse study shows gene therapy may be possible cure for Hurler syndrome

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PUBLIC RELEASE DATE:

4-Feb-2014

Contact: Nick Miller nicholas.miller@cchmc.org 513-803-6035 Cincinnati Children's Hospital Medical Center

CINCINNATI Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome an often fatal condition that causes organ damage and other medical complications.

Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and Stroke (NINDS) report their unique strategy for treating the disease the week of Feb. 3-7 in Proceedings of the National Academy of Sciences (PNAS).

Researchers were able to genetically insert into the cells a gene that produces a critical lysosomal enzyme (called IDUA) and then inject the engineered cells into mice to treat the disorder. Follow up tests showed the treatment resulted in a complete metabolic correction of the disease, according to the authors.

"Our findings demonstrate a unique and somewhat surprising delivery pathway for lysosomal enzymes," said Dao Pan, PhD, corresponding author and researcher in the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's. "We show proof of concept that platelets and megakaryocytes are capable of generating and storing fully functional lysosomal enzymes, which can lead to their targeted and efficient delivery to vital tissues where they are needed."

The mice tested in the study modeled human Hurler syndrome, a subset of disease known as mucopolysaccharidosis type I (MPS I), one of the most common types of lysosomal storage diseases. MPS I is a lysosomal storage disease in which people do not make an enzyme called lysosomal alpha-L-iduronidase (IDUA).

IDUA helps break down sugar molecules found throughout the body, often in mucus and fluids around joints, according to the National Library of Medicine/National Institutes of Health. Without IDUA, sugar molecules build up and cause organ damage. Depending on severity, the syndrome can also cause deafness, abnormal bone growth, heart valve problems, joint disease, intellectual disabilities and death.

Enzyme replacement therapy can be used to treat the disease, but it is only temporary and not curative. Bone marrow transplant using hematopoietic stem cells also has been tested on some patients with mixed results. The transplant procedure can carry severe risks and does not always work.

Continued here:

Mouse study shows gene therapy may be possible cure for Hurler syndrome