Archive for May, 2012

OLDSMAR, Fla., May 3, 2012 (GLOBE NEWSWIRE) -- via PRWEB - Cryo-Cell International, Inc. announced the appointment of Linda Kelley, Ph.D., as chief scientific officer. Dr. Kelley is responsible for overseeing Cryo-Cells state-of-the art laboratory, translational medicine initiatives and quality assurance program at its stem cell and cord blood banking facility in Oldsmar, Florida. She joins the company from the Dana Farber Cancer Institute at Harvard, where she was the director of the Connell OReilly Cell Manipulation Core Facility.

Dr. Kelley is an internationally recognized, cord blood stem cell scientist whose accomplishments have helped expand the scope of stem cell therapies from bone marrow transplantation to the treatment of heart, kidney, brain and other degenerative diseases. She was a member of the board of trustees of the Foundation for Accreditation of Cellular Therapy and chaired its Standards Committee. Dr. Kelley was one of 12 scientists selected by the Institute of Medicine of the National Academies of Science to serve on the panel that advised Congress on how to allocate $80 million in funding to optimally structure a national cord blood stem cell program.

While director of the Cell Therapy Facility at the University of Utah, she established that states first umbilical cord blood collection program that enabled families to donate their childrens cord blood to the national inventory. Dr. Kelley earned graduate and post-doctoral degrees in hematology and immunology at Vanderbilt University in Nashville, Tenn., where she also served as assistant professor in the Department of Medicine.

As a leader in our field, Cryo-Cell is delighted to have someone of Dr. Kelleys caliber directing our laboratory and translational medicine initiatives. Her expertise will ensure that we continue to exceed the industrys quality standards and maintain our tradition of offering clients the absolute best in cord blood, cord tissue, and menstrual stem cell cryopreservation services, said David Portnoy, chairman and co-CEO at Cryo-Cell. Under her guidance, Cryo-Cell will be propelled to the forefront of regenerative medicine.

Kelley replaces Julie Allickson, Ph.D., who is joining the Wake Forest Institute for Regenerative Medicine (WFIRM), where she will manage translational research. WFIRM is led by Anthony Atala, M.D., a Cryo-Cell board member and preeminent stem cell scientist.

The opportunity to work in a cutting-edge facility with a staff that is exceptionally well trained was very attractive to me, said Dr. Kelley. But equally important in my decision to join Cryo-Cell, was the commitment that co-CEOs David and Mark Portnoy have made to support the advancement of regenerative medicine through partnerships with Stanford University and private research facilities. Cryo-Cell is unique among stem cell cryopreservation firms in that regard.

About Cryo-Cell International, Inc. Cryo-Cell International, Inc. was founded in 1989. In 1992, it became the first private cord blood bank in the world to separate and store stem cells. Today, Cryo-Cell has over 240,000 clients worldwide from 87 countries. Cryo-Cell's mission is to provide clients with state-of-the-art stem cell cryopreservation services and support the advancement of regenerative medicine. Cryo-Cell operates in a facility that is compliant with Good Manufacturing Practice and Good Tissue Practice (cGMP/cGTP). It is ISO 9001:2008 certified and accredited by the American Association of Blood Banks. Cryo-Cell is a publicly traded company, OTC:QB Markets Group Symbol: CCEL. Expectant parents or healthcare professionals who wish to learn more about cord blood banking and cord blood banking prices may call 1-800-STOR-CELL (1-800-786-7235) or visit http://www.cryo-cell.com/.

Forward-Looking Statement Statements wherein the terms "believes", "intends", "projects", "anticipates", "expects", and similar expressions as used are intended to reflect "forward-looking statements" of the Company. The information contained herein is subject to various risks, uncertainties and other factors that could cause actual results to differ materially from the results anticipated in such forward-looking statements or paragraphs, many of which are outside the control of the Company. These uncertainties and other factors include the success of the Company's global expansion initiatives and product diversification, the Company's actual future ownership stake in future therapies emerging from its collaborative research partnerships, the success related to its IP portfolio, the Company's future competitive position in stem cell innovation, future success of its core business and the competitive impact of public cord blood banking on the Company's business, the Company's ability to minimize future costs to the Company related to R&D initiatives and collaborations and the success of such initiatives and collaborations, the success and enforceability of the Company's menstrual stem cell technology license agreements and umbilical cord blood license agreements and their ability to provide the Company with royalty fees, the ability of the reproductive tissue storage to generate new revenues for the Company and those risks and uncertainties contained in risk factors described in documents the Company files from time to time with the Securities and Exchange Commission, including the most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and any Current Reports on Form 8-K filed by the Company. The Company disclaims any obligations to subsequently revise any forward-looking statements to reflect events or circumstances after the date of such statements.

Contact: David Portnoy Cryo-Cell International, Inc. 813-749-2100 dportnoy(at)cryo-cell(dot)com

This article was originally distributed on PRWeb. For the original version including any supplementary images or video, visit http://www.prweb.com/releases/2012/5/prweb9469228.htm

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Cryo-Cell International Taps Leader in Stem Cell Therapy to Serve as Chief Scientific Officer

03-05-2012 13:31 A study from Professor Franck Polleux's lab at The Scripps Research Institute has shown that an extra copy of a brain-development gene allowed neurons to migrate farther and develop more connections as human evolution progressed. The findings, published in the journal Cell, may offer a clue to autism and schizophrenia.

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Scripps Research Scientists Show How a Gene Duplication Helped our Brains Become 'Human' - Video

May 4, 2012

Brett Smith for Redorbit.com

Scientists may have just found a missing link in the form of a partial, duplicate gene that appears to be responsible for human brain development the most distinguishing characteristic of our species.

The genetic variation occurred in mans ape-like ancestor about two or three million years ago, according to a pair of studies published online in the journal Cell. A team led by researchers at the Scripps Research Institute found that a partial SRGAP2 gene duplicates interfere with the function of the original gene and allows maturing neurons to migrate farther and develop more connections.

This appears to be a major example of a genomic innovation that contributed to human evolution, said Franck Polleux, a professor at The Scripps Research Institute.

The SRGAP2 gene was first singled out for study by researchers because it helps drive development of the neocortex, which controls higher-order brain functions. Mutations in this gene have been found to cause certain brain disorders.

Another group of researchers led by Evan Eichler of the University of Washington discovered that SRGAP2 duplicated itself 3.5 million years ago, after humans and chimps diverged. One million years later, this partial copy, or daughter, of the original gene underwent its own duplication and created a granddaughter copy. Like a game of Telephone, each version of the gene underwent certain changes so that they resembled the original less and less with each successive copy.

These evolutionarily recent gene duplications are so nearly identical to the original genes that they arent detectable by traditional genome sequencing methods, said Polleux. Only in the last five years have scientists developed methods to reliably map these hominid-specific duplications.

To test their theory, Polleux and his colleagues put human copies of the daughter and granddaughter SRGAP2 genes into mice. The proteins made by these human genes bound to the original SRGAP2 and hindered the genes ability to do its job.

Although the mouse didnt develop a human brain, the neurons in the neocortex grew to look like human brain cells. The neurons also formed 50 to 60 percent more of these spines than normal mouse neurons, a sign of higher brain power.

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Human Brain Evolution Triggered By Duplicate Gene

ScienceDaily (May 3, 2012) Researchers at Weill Cornell Medical College and Rockefeller University have identified the first gene to be linked to a rare condition in which babies are born without a spleen, putting those children at risk of dying from infections they cannot defend themselves against. The gene, Nkx2.5, was shown to regulate genesis of the spleen during early development in mice.

The study, published online May 3 in Developmental Cell, raises the hope that a simple genetic screening test for Nkx2.5 mutations can be developed that will alert parents that their developing child may be missing the organ, which could then be confirmed with a diagnostic scan.

"The great news is that with the appropriate preventive antibiotic treatment these children will not succumb to fatal infections. This test could potentially save lives," says the study's lead investigator, Dr. Licia Selleri, an associate professor in the Department of Cell and Developmental Biology at Weill Cornell Medical College.

Because defense against infections depends, in part, on the spleen, children known to be born without the organ require treatment with a regimen of antibiotic therapy throughout their lives. But most diagnoses of this condition, congenital asplenia, are made during an autopsy after a child dies, suddenly and unexpectedly, from a rapidly lethal infection, usually from bacteria that causes pneumonia or meningitis, Dr. Selleri says. "For those reasons, we believe this condition is not quite as rare as believed. Not every child who dies from an infection is given an autopsy."

Long search for genetic culprits

Patients with congenital asplenia usually lack a spleen as the sole abnormality, but sometimes have abnormalities of the heart and blood vessels. The majority of those cases arise sporadically, so are not believed to be inherited. One form of this disorder is known as Isolated Congenital Asplenia (ICA), characterized by a spleen that is missing but with no other developmental abnormalities. The cause is believed to be genetic, but no candidate genes in humans had been found before this study.

This research project was a collaboration between Dr. Selleri and her colleagues, and Rockefeller University's Dr. Jean-Laurent Casanova, professor in the St. Giles Laboratory of Human Genetics of Infectious Diseases. Dr. Casanova had led a previous study describing 20 ICA patients, of which most children suffered their first serious infection by age one, and nine died of an invasive pneumonia.

Dr. Selleri has long been studying congenital asplenia in the laboratory using the mouse as a model system and had previously discovered that a transcription factor known as Pbx is the prime regulator of spleen development in mouse models. Dr. Matthew Koss, a recent Ph.D. graduate who had studied in Dr. Selleri's lab, led the effort to create a strain of mice that lacked Pbx in the spleen, and were born without a spleen. He identified a regulatory module that is controlled by Pbx and targets Nkx2.5, a gene downstream of Pbx, in the developing spleen of the mouse embryo. He also discovered that Pbx controls the growth of the spleen by directly regulating the expression of Nkx2.5, which in turn controls cell proliferation within the primitive spleen organ.

Then, in Dr. Casanova's lab, Alexandre Bolze, a graduate student, sequenced genetic samples from ICA patients and analyzed them using whole exome sequencing technology, which allows sequencing of the entire coding genome of multiple patients -- a technique routinely employed by Dr. Casanova. Bolze found that Nkx2.5 was mutated in a family of asplenic patients, some of which died from lethal infections -- confirming the importance of Nkx2.5 in human congenital asplenia as in the mouse model of the disorder.

"This study illustrates the unique strength in using mouse models and human genetics hand-in-hand," says Dr. Selleri. "It demonstrates how genetic pathways identified in mouse models can be exploited to further understand the pathogenesis of human disease towards a better prenatal diagnosis."

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First gene linked to missing spleen in newborns

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

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

SAN DIEGO, May 3, 2012 /PRNewswire/ --Sequenom, Inc. (SQNM), a life sciences company providing innovative genetic analysis solutions, today reported revenue of $14.9 million for the first quarter of 2012, an increase of 10% compared to revenue of $13.5 million for the first quarter of 2011. First quarter 2012 revenues from the Sequenom Center for Molecular Medicine (Sequenom CMM) diagnostics services operating segment grew more than 187%, while revenues from the genetic analysis operating segment decreased 14% in the first quarter of 2012 as compared to the prior year period. Net loss for the first quarter of 2012 was $24.5 million, or $0.22 per share, as compared to net loss of $12.7 million, or $0.13 per share for the same period in 2011, reflecting an increase in costs associated primarily with the early commercialization of the Sequenom CMM MaterniT21 PLUS laboratory developed test (LDT).

As to be expected in the first year following the launch of a new LDT, gross margin was reduced in the first quarter of 2012 as compared to the same period one year ago, falling to 36% of revenue as compared to gross margin of 63% for the first quarter of 2011. Diagnostic services revenue is primarily recognized when cash is received and costs are recognized at the time services are performed. The delay in revenue recognition results in a decrease in gross margin, which is significantly magnified by increasing test volumes. Therefore, diagnostic revenue recognized in the current period does not relate directly to the costs incurred in the same period. This will continue until such time as the company converts to accrual accounting for diagnostic services revenue which is expected when sufficient reimbursement history has been established.

Gross margin is expected to continue to fluctuate on a quarterly basis due primarily to changes in sales volumes and the timing of cash receipts. During the first quarter of 2012, gross margin on diagnostic services revenue was negative, while gross margin on genetic analysis revenues increased by 3% to 74%, primarily due to a change in product mix, with higher margin consumables comprising a larger percentage of overall segment revenues.

Total operating expenses for the first quarter of 2012 were $29.8 million, as compared to total expenses of $21.6 million for the first quarter of 2011. This change reflects increased selling and marketing expenses resulting primarily from higher labor costs associated with increased headcount and an expanded diagnostic sales force, as well as increased research and development expenses associated with facilities allocation and overhead, higher operational supply expenses and labor related costs.

General and administrative expenses also increased for the first quarter, primarily due to increased facilities and legal expenses as compared to the same period one year ago. Total stock-based compensation expense was $2.9 million for the first quarter of 2012, as compared to $2.6 million as recorded during the first quarter in 2011.

"Our performance during the quarter reflects the successful achievement of our operational goals throughout the organization, specifically those associated with the expansion of Sequenom CMM's testing services and the launch and rapid adoption of the MaterniT21 PLUS laboratory developed test," said Paul V. Maier, Sequenom's CFO. "We ended the quarter with a favorable cash position as we continued to strengthen our balance sheet, raising $58.2 million during the period through an underwritten public offering, the proceeds of which will primarily be used to fund the expansion of our operations in support of the growing demand for our testing services."

As of March 31, 2012, total cash, cash equivalents, and marketable securities were $119.7 million. Net cash used in operating activities was $23.3 million for the first quarter, while purchases of capital equipment for the same period totaled $1.8 million, funded primarily through utilization of the Company's credit facility. As of March 31, 2012, the Company had borrowed $17.0 million under the credit facility.

Operational Updates

The Company recently reported that more than 12,700 total tests, including in excess of 4,900 MaterniT21 PLUS laboratory-developed tests (LDT) offered by Sequenom CMM, were accessioned during the first quarter of 2012, a 140% increase over the same period in 2011.At the close of the first quarter of 2012, the number of MaterniT21 PLUS samples accessioned on an annualized basis equated to approximately 30,000 tests. As of the last week in April, the52-week runrate had increased to more than 45,000 tests.

"The first quarter of 2012 has been an active and productive one for Sequenom and Sequenom CMM, as we have received and responded to the strong interest in Sequenom CMM's testing services, particularly with regard to Sequenom CMM's MaterniT21 PLUS LDT," said Harry Hixson, Jr., Ph.D., Chairman and CEO of Sequenom. "It is our goal to build on this positive momentum as the year progresses. We will continue to expand our reach to meet growing demand and deliver on our objective of providing high quality testing services offered by Sequenom CMM to physician customers nationwide."

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Sequenom, Inc. Reports Financial Results For The First Quarter Of 2012

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

Excess sun exposure, a diet rich in fish, and gene inheritance from ancient explorers and traders, are all possible theories why some dark-skinned indigenous Solomon Islanders are naturally blonde, according to new research published today in the journal Science.

The study, led by Stanford University researchers, found that 5 to 10 percent of the indigenous Solomon population have a gene that is responsible for blondeness. The trait, however, is distinctly different from the gene that causes blond hair in Europeans. Their findings reveal a genetic variant which has led the islanders to have simultaneously the darkest skin pigmentation outside of Africa and the highest prevalence of blonde hair outside of Europe.

Previous studies have proven that pigmentation is largely genetic but also has evolved to adapt to the Suns ultraviolet rays with populations near the equator having the darkest skin and hair color. However, the native Solomon Islanders differ from this trend.

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

The research, co-led by researchers at Stanford University and Dr. Nic Timpson from the University of Bristol, sought out to find why these islanders possess such strikingly dissimilar hair and skin patterning in the world.

For the study, the team took samples from a pool of more than 1,000 Melanesian participants, 43 of which had blonde hair and 42 of which had dark hair. They carried out genetic analyses on the samples to compare their genomes. The results showed that across the whole genome, one key gene area contained the gene variation TYRP1 responsible for cell differences that produce dark pigmentation.

TYRP1 is known to influence pigmentation in humans. But the researchers found the variant of TYRP1 that causes the blonde hair in Solomon Islanders is entirely absent from the genomes in Europeans.

Here you go into an unstudied population with a small sample size and you can really find some cool things, said study coauthor Carlos Bustamante, a geneticist at Stanford Universitys School of Medicine. So what about other places, like what about light pigmentation in parts of Africa? How do we not know the genetic basis of skin and hair pigmentations across the globe?

Naturally blonde hair is a surprisingly unusual trait in humans which is typically associated with people from Scandinavian and Northern European countries, said Timpson. Our findings help explain the fascinating differences in these physical characteristics, but also underline the importance of genetic mapping using isolated populations to help shed new light on the epidemiology of disease.

Many experts believed the blonde hair of Melanesia was the result of a trait passed on by Europeans who visited the islands centuries ago.

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Blondeness In Solomon Islanders Due To Genetic Variations

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

(CBS News) Love meat? Hate meat? The reason for your answer may come down to genes. In a new study, scientists investigated whether people with a stronger sensitivity to the smell of pork are more likely to be meat-eaters.

Flavor is a combination of factors including taste and smell. According to the study, some people have receptors that detect a steroid called androstenone, which is found in high concentrations in male pigs - and in turn pork. Most commercially raised animals in the U.S. are actually castrated to get rid of the smell, but previous research has found that people who have two copies of the gene that helps sense androstenone still smell the odor - and might have a mixed reaction to pork.

"For those who are very sensitive to it, it's really disgusting. It's a sweaty, urine-like odor," Dr. Hiroaki Matsunami, an associate professor of molecular genetics and microbiology at Duke University in Durham, N.C. , told WebMD. "For others, you can smell it, but it's not as bad. Those people say it smells fragrant, chemical, or sweet."

For the study, published in the journal PLoS ONE, researchers examined the difference between people with one copy of the gene that helps detect androstenone and those with two. The study had 23 participants - 13 regular eaters and 10 "professional sensory assessors" who have sensitive noses and are trained to pick up certain scents. Participants were exposed to androstenone daily for six weeks, and then asked to evaluate samples of cooked meat with different levels of androstenone.

The participants rated the taste and smell of the meat - and then the researchers tested their genes. What did they find? Participants who rated the flavor of pork as bad were more likely to have two copies of the gene that picks up the scent.

"I was surprised at how cleanly this experiment showed who smelled what," Matsunami said in a university news release. "The results showed that people with two copies of the functional variant of the gene for that odor receptor thought that the meat smelled worse with higher levels of androstenone added."

These findings suggest that vegetarians may have a genetic predisposition against meat, according to Matsunami.

What do you think of the smell of meat?

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Genetics may explain why some people hate meat, study says

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 ...

LOS ANGELES--(BUSINESS WIRE)--

Response Genetics, Inc. (Nasdaq:RGDX - News),a company focused on the development and commercialization of molecular diagnostic tests for cancer, will announce its first quarter financial results and an operational update in a press release to be issued before the market opens on Thursday, May 10, 2012. The company will host a conference call that same day at 10:00 a.m. EDT to discuss its financial results.

CONFERENCE CALL DETAILS

To access the conference call by phone on May 10 at 10:00 a.m. EDT, dial (800) 537-0745 or (253) 237-1142 for international participants. A telephone replay will be available beginning approximately two hours after the call through May 17, and may be accessed by dialing (855) 859-2056, (404) 537-3406, or (800) 585-8367. The replay passcode is 77936576.

To access the live and archived webcast of the conference call, go to the Investor Relations section of the Company's Web site at http://investor.responsegenetics.com. It is advised that participants connect at least 15 minutes prior to the call to allow for any software downloads that might be necessary.

About Response Genetics, Inc.

Response Genetics Inc. (RGI) is a CLIA-certified clinical laboratory focused on the development and sale of molecular diagnostic tests for cancer. RGIs principal customers include oncologist, pathologists and hospitals. In addition to diagnostic testing services, the Company generates revenue from the sales of its analytical testing services of clinical trial specimens to the pharmaceutical industry. RGI was founded in 1999 and its principal headquarters are located in Los Angeles, California. For additional information, please visit http://www.responsegenetics.com.

Forward-Looking Statement Notice

Except for the historical information contained herein, this press release and the statements of representatives of RGI related thereto contain or may contain, among other things, certain forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995.

Such forward-looking statements involve significant risks and uncertainties. Such statements may include, without limitation, statements with respect to the Companys plans, objectives, projections, expectations and intentions, such as the ability of the Company to announce its financial results and provide a conference call, to continue to provide clinical testing services to the medical community, to continue to expand its sales force, to continue to build its digital pathology initiative, to attract and retain qualified management, to continue to provide clinical trial support to pharmaceutical clients, to enter into new collaborations with pharmaceutical clients, to enter into new areas such as companion diagnostics, and to continue to execute on its business strategy and operations, to continue to analyze cancer samples, the potential for using the results of this research to develop diagnostic tests for cancer, the usefulness of genetic information to tailor treatment to patients, and other statements identified by words such as projects, may, could, would, should, believes, expects, anticipates, estimates, intends, plans or similar expressions.

Read the original:
Response Genetics, Inc. to Release First Quarter Financial Results and Host Conference Call on May 10, 2012

By Randy Dotinga HealthDay Reporter

WEDNESDAY, May 2 (HealthDay News) -- New research shows that gene therapy can have long-lasting effects on the immune cells of HIV patients -- a promising sign -- even though the specific treatment being studied did not eradicate the virus.

This approach is one of several gene therapy strategies that are being investigated by scientists as possible ways to keep the AIDS virus from spreading in the blood.

In this case, "people were treated by gene therapy and nothing bad happened. It was safe," said study co-author Frederic Bushman, a professor of microbiology at the University of Pennsylvania.

In addition, he said, the treated immune cells managed to remain around for about a decade. "The general picture that emerges about genetic alterations to human immune cells is that they can persist for a long time if you do it right."

The study appears in the May 2 issue of Science Translational Medicine.

Researchers have long been exploring gene therapy -- in which cells in the body are genetically modified -- as a possible treatment for infection with HIV, the virus that causes AIDS. The idea is that the therapy would offer a permanent alternative to costly medications that come with potentially disabling side effects.

"Just think about what an HIV patient has to do: take drugs every day for the rest of his life, and the minute he stops taking them, the virus starts coming back," said John Rossi, chair of the department of molecular and cellular biology at the Beckman Research Institute of the City of Hope, in Duarte, Calif. He was not associated with the new research.

The study looks at 43 HIV-positive patients. Between 1998 and 2002, researchers removed blood from the patients, genetically modified it, and injected it back into them.

The plan was to program immune cells known as T cells to kill HIV cells.

Read more:
Gene therapy for HIV safe, but effectiveness still unclear

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

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

CINCINNATI Researchers have rejuvenated aged hematopoietic stem cells to be functionally younger, offering intriguing clues into how medicine might one day fend off some of the ailments of old age.

Scientists at Cincinnati Children's Hospital Medical Center and the Ulm University Medicine in Germany report their findings online May 3 in the journal Cell Stem Cell. The paper brings new perspective to what has been a life science controversy countering what used to be broad consensus that the aging of hematopoietic stem cells (HSCs) was locked in by nature and not reversible by therapeutic intervention.

HSCs are stem cells that originate in the bone marrow and generate all of the body's red and white blood cells and platelets. They are an essential support mechanism of blood cells and the immune system. As humans and other species age, HSCs become more numerous but less effective at regenerating blood cells and immune cells. This makes older people more susceptible to infections and disease, including leukemia.

Researchers in the current study determined a protein that regulates cell signaling Cdc42 also controls a molecular process that causes HSCs from mice to age. Pharmacologic inhibition of Cdc42 reversed HSC aging and restored function similar to that of younger stem cells, explained Hartmut Geiger, PhD, the study's principal investigator and a researcher in the Division of Experimental Hematology/Cancer Biology at Cincinnati Children's, and the Department of Dermatology and Allergic Diseases, Ulm University Medicine.

"Aging is interesting, in part because we still don't understand how we age," Geiger said. "Our findings suggest a novel and important role for Cdc42 and identify its activity as a target for ameliorating natural HSC aging. We know the aging of HSCs reduces in part the response of the immune system response in older people, which contributes to diseases such as anemia, and may be the cause of tissue attrition in certain systems of the body."

The findings are early and involve laboratory manipulation of mouse cells, so it remains to be seen what direct application they may have for humans. Still, the study expands what is known about the basic molecular and cellular mechanisms of aging a necessary step to one day designing rational approaches to aiding a healthy aging process.

One reason the research team focused on Cdc42 is that previous studies have reported elevated activity of the protein in various tissue types of older mice which have a natural life span of around two years. Also, elevated expression of Cdc42 has been found in immune system white blood cells in older humans.

In the current study, researchers found elevated activity of Cdc42 in the HSCs of older mice. They also were able to induce premature aging of HSCs in mice by genetically increasing Cdc42 activity in the cells. The aged cells lost structural organization and polarity, resulting in improper placement and spacing of components inside the cells. This disorganization contributed to the cells' decreased functional efficiency.

Excerpt from:
Aged hematopoietic stem cells rejuvenated to be functionally younger

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that Massachusetts Eye and Ear (Mass. Eye and Ear) has received institutional review board (IRB) approval to be a site for the companys Phase I/II clinical trial for dry age-related macular degeneration (dry AMD), using human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells.

We are delighted to announce that Mass. Eye and Ear will participate as a site for our clinical trial for dry AMD, said Gary Rabin, ACTs chairman and CEO. Dr. Dean Eliott and his team are deeply committed to finding new treatments for preventing blindness, and we very much look forward to tapping into his expertise and insight into the progression of macular degenerative disorders. The primary teaching hospital for ophthalmology at Harvard Medical School, Mass. Eye and Ear is ranked as among the top ophthalmology hospitals in the country by U.S. News & World Report and has a reputation that is unrivaled.

The Phase I/II trial is a prospective, open-label study designed to determine the safety and tolerability of the hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD. The trial will ultimately enroll 12 patients, with cohorts of three patients each in an ascending dosage format.

Dry AMD represents one of the largest unmet medical needs in ophthalmology, commented Dr. Dean Eliott, M.D. a full time retina surgeon, scientist and Associate Director of the Retina Service at Mass. Eye and Ear. We appreciate the opportunity to get some first-hand experience with the protocol and be involved with the international team that has been assembled around the U.S. and European trials.

Founded in 1824, the Massachusetts Eye and Ear Infirmary is an independent specialty hospital affiliated with Harvard Medical School.

Further information about patient eligibility for the dry AMD study is available at http://www.clinicaltrials.gov; ClinicalTrials.gov Identifier: NCT01344993.

About dry AMD Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. Age-related macular degeneration (AMD) is the leading cause of blindness in people over age 60 in the United States, and the vast majority of cases of AMD are of the dry form, which is currently untreatable.

About hESC-derived RPE Cells The retinal pigment epithelium (RPE) is a highly specialized tissue located between the choroid and the neural retina. RPE cells support, protect and provide nutrition for the light-sensitive photoreceptors. Human embryonic stem cells differentiate into any cell type, including RPE cells, and have a similar expression of RPE-specific genes compared to human RPE cells and demonstrate the full transition from the hESC state.

About Advanced Cell Technology, Inc. Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

More here:
ACT Announces Massachusetts Eye and Ear as Additional Site for Clinical Trial for Dry Age-Related Macular Degeneration ...

PHILADELPHIA HIV patients treated with genetically modified T cells remain healthy up to 11 years after initial therapy, researchers from the Perelman School of Medicine at the University of Pennsylvania report in the new issue of Science Translational Medicine. The results provide a framework for the use of this type of gene therapy as a powerful weapon in the treatment of HIV, cancer, and a wide variety of other diseases.

"We have 43 patients and they are all healthy," says senior author Carl June, MD, a professor of Pathology and Laboratory Medicine at Penn Medicine. "And out of those, 41 patients show long term persistence of the modified T cells in their bodies."

Early gene therapy studies raised concern that gene transfer to cells via retroviruses might lead to leukemia in a substantial proportion of patients, due to mutations that may arise in genes when new DNA is inserted. The new long-term data, however, allay that concern in T cells, further buoying the hope generated by work June's team published in 2011 showing the eradication of tumors in patients with chronic lymphocytic leukemia using a similar strategy.

"If you have a safe way to modify cells in patients with HIV, you can potentially develop curative approaches," June says. "Patients now have to take medicine for their whole lives to keep their virus under control, but there are a number of gene therapy approaches that might be curative." A lifetime of anti-HIV drug therapy, by contrast, is expensive and can be accompanied by significant side effects.

They also note that the approach the Penn Medicine team studied may allow patients with cancers and other diseases to avoid the complications and mortality risks associated with more conventional treatments, since patients treated with the modified T cells did not require drugs to weaken their own immune systems in order for the modified cells to proliferate in their bodies after infusion, as is customary for cancer patients who receive stem cell transplants.

To demonstrate the long-term safety of genetically modified T cells, June and colleagues have followed HIV-positive patients who enrolled in three trials between 1998 and 2002. Each patient received one or more infusions of their own T cells that had been genetically modified in the laboratory using a retroviral vector. The vector encoded a chimeric antigen receptor that recognizes the HIV envelope protein and directs the modified T cell to kill any HIV-infected cells it encounters.

As is standard for any trial, the researchers carefully monitored patients for any serious adverse events immediately after infusion -- none of which were seen. Additionally, because of the earlier concerns about long-term side effects, the U.S. Food and Drug Administration also asked the team to follow the patients for up to 15 years to ensure that the modified T cells were not causing blood cancers or other late effects. Therefore, each patient underwent an exam and provided blood samples during each of the subsequent years.

Now, with more than 500 years of combined patient safety data, June and colleagues are confident that the retroviral vector system is safe for modifying T cells. By contrast, June notes, the earlier, worrying side effects were seen when viral vectors were used to modify blood stem cells. The new results show that the target cell for gene modification plays an important role in long-term safety for patients treated. "T cells appear to be a safe haven for gene modification," June says.

The multi-year blood samples also show that the gene-modified T cell population persists in the patients' blood for more than a decade. In fact, models suggest that more than half of the T cells or their progeny are still alive 16 years after infusion, which means one treatment might be able to kill off HIV-infected cells for decades. The prolonged safety data means that it might be possible to test T cell-based gene therapy for the treatment of non-life threatening diseases, like arthritis.

"Until now, we've focused on cancer and HIV-infection, but these data provide a rationale for starting to focus on other disease types," June says. "What we have demonstrated in this study and recent studies is that gene transfer to T cells can endow these cells with enhanced and novel functions. We view this as a personalized medicine platform to target disease using a patient's own cells."

Read more from the original source:
Genetically Modified T Cell Therapy Shown to be Safe, Lasting in Decade-Long Penn Medicine Study of HIV Patients

EXTON, Pa.--(BUSINESS WIRE)--

In collaboration with Fibrocell Science, Inc., (OTCBB:FCSC.OB), researchers at the University of California, Los Angeles (UCLA) have identified two rare adult stem cell-like subpopulations in adult human skin, a discovery that may yield further ground-breaking research in the field of personalized medicine for a broad range of diseases. Using technology developed by Fibrocell Science, Inc. the researchers were able to confirm the existence of these two types of cells in human skin cell cultures, potentially providing a source of stem cell-like subpopulations from skin biopsies, which are quicker to perform, relatively painless and less invasive than bone marrow and adipose tissue extractions, which are the current methods for deriving adult stem cells for patient-specific cellular therapies.

The findings, which are reported in the inaugural issue of BioResearch Open Access, pertain to two subtypes of cells: SSEA3-expressing regeneration-associated (SERA) cells, which may play a role in the regeneration of human skin in response to injury and mesenchymal adult stem cells (MSCs), which are under investigation (by many independent researchers) for their ability to differentiate into the three main types of cells: osteoblasts (bone cells), chondrocytes (cartilage cells) and adipocytes (fat cells). Finding these specialized cells within the skin cell cultures is important because rather than undergoing a surgical organ or tissue transplantation to replace diseased or destroyed tissue, patients may one day be able to benefit from procedures by which stem cells are extracted from their skin, reprogrammed to differentiate into specific cell types and reimplanted into their bodies to exert a therapeutic effect. Research in this area is ongoing.

Finding these rare adult stem cell-like subpopulations in human skin is an exciting discovery and provides the first step towards purifying and expanding these cells to clinically relevant numbers for application to a variety of potential personalized cellular therapies for osteoarthritis, bone loss, injury and/or damage to human skin as well as many other diseases, said James A. Byrne, Ph.D., the studys lead author and Assistant Professor of Molecular and Medical Pharmacology at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. In addition to pursuing our own research investigations with Fibrocell Science using this method, we envision a time not too far in the future when we will be able to isolate and produce mesenchymal stem cells and SERA cells on demand from skin samples, which may allow other researchers in need of specialized cells to pursue their own lines of medical and scientific research.

We congratulate the UCLA researchers on the publication of their breakthrough data, which may ultimately lead to new patient-specific, personalized cellular therapies to treat various diseases, said David Pernock, Chairman and CEO of Fibrocell Science, Inc. Fibrocell Science is proud of our role in helping to establish the potential of dermal skin cells for the future of personalized, regenerative medicine. We look forward to continuing our relationship with UCLA and Dr. Byrnes team to advance this research.

Discovering Viable, Regenerative Cells in the Skin

Dr. Byrne and colleagues confirmed previous research identifying a rare population of cells in adult human skin that has a marker called the stage-specific embryonic antigen 3 (SSEA3). Dr. Byrne observed that there was a significant increase in the number of SSEA3 expressing cells following injury to human skin, supporting the hypothesis that the SSEA3 biomarker can be used to facilitate the identification and isolation of these cells with tissue-regenerative properties.

Using Fibrocells proprietary technology, the researchers collected cells from small skin samples, cultured the cells in the lab, and purified them via a technique known as fluorescence-activated cell sorting (FACS). Under FACS, cells in suspension were tagged with fluorescent markers specific for undifferentiated stem cells. This method allowed the researchers to separate the rare cell subpopulations from other types of cells.

Dr. Byrne and colleagues also observed a rare subpopulation of functional MSCs in human skin that existed in addition to the SERA cells.

Being able to identify two sub-populations of rare, viable and functional cells that behave like stem cells from within the skin is an important finding because both cell types have the potential to be investigated for diverse clinical applications, said Dr. Byrne.

See more here:
Fibrocell Science Technology Leads to Discovery of Two Rare Adult Stem Cell-Like Subpopulations in Human Skin

SAN DIEGO, CA--(Marketwire -05/03/12)- Regen BioPharma (Regen), Inc. a newly-formed subsidiary of Bio-Matrix Scientific Group, Inc. (BMSN.PK - News) (BMSN.PK - News), unveiled today its operational plan for its "Super-Incubator" stem cell company.

Month 1-2: Assembly of Team. Regen intends to assemble a team of world-class leaders in the spheres of Technology, Intellectual Property assessment, valuation and Clinical development. Regen will seek to compile a team of Physician-Scientists with experience in the area of clinical trials for regenerative medicine/stem cell products, Regulatory experts who have successfully taken products through the FDA and corresponding agencies internationally, and Biotech Entrepreneurs who have track records of excellence in business formation and value optimization.

Month 1-4: In-licensing of Intellectual Property. The Company having already assessed over 20,000 issued patents and having compiled a shortlist of 30 targets; Regen will seek to execute licensing deals on an initial core of 3 technologies. Regen focuses on issued patents that have already passed preclinical studies but are not under clinical development.

Month 3-6: Interaction with Regulatory Agencies. Regen intends to develop data packages for each of the technologies and initiate interaction with Regulatory Agencies such as the FDA for initiation of trials.

Month 6-18: Clinical Implementation. Regen intends to launch clinical trials with world-leading institutions to obtain human safety data and "signal" of therapeutic efficacy.

Month 18-24: Exit. It is intended that technologies "incubated" by Regen will be spun off either as separate companies, or sold to Large Pharma companies seeking to enhance their therapeutic pipeline.

"At present there exists a wealth of intellectual property that is 'collecting dust' in the corridors of Academia. Given the field of regenerative medicine and stem cell therapy is so young, and the business models are fuzzy at best in terms of valuation, we see this space as a unique opportunity for acceleration of clinical development/value optimization," said Bio-Matrix Chairman & CEO David Koos about its Regen BioPharma. "Valuations for stem cell companies that have passed the threshold of clinical safety, with signals of efficacy are astronomical. The $1.8 billion Mesoblast-Cephalon deal, as well as recent financings of private companies with as little as 3 patient data such as Promethera ($31 million) or Allocure with 16 patients ($23 million), is testimony to the extremely high valuations that are characteristic of this space."

About Bio-Matrix Scientific Group, Inc.:

Bio-Matrix Scientific Group, Inc. (BMSN.PK - News) is a biotechnology company focused on the development of regenerative medicine therapies and tools. The Company is specifically focused on human therapies that address unmet medical needs. Specifically, Bio-Matrix Scientific Group Inc. is looking to increase the quality of life through therapies involving stem cell treatments. These treatments are focused in areas relating to lung, heart, circulatory system and other internal organs.

Through Its wholly owned subsidiary, Regen BioPharma, it is the Company's goal to develop translational medicine platforms for the rapid commercialization of stem cell therapies. The Company is looking to use these translational medicine platforms to advance intellectual property licensed from entities, institutions and universities that show promise towards fulfilling the Company's goal of increased quality of life.

View post:
Bio-Matrix Subsidiary "First in Class" Approach to Stem Cell Medicine

"See The Potential" program sponsored by Canada's Stem Cell Network and Pfizer

MONTRAL, May 2, 2012 /CNW/ - The first two postdoctoral research fellowships of a new program to promote stem cell research in Canada were announced today by the program's sponsors, Canada's Stem Cell Network and Pfizer.

"See The Potential" is a program established to encourage the work of promising young scientists in the field of stem cell and regenerative medicine research. Under the program, six postdoctoral fellowships will be funded from competitions over the next three years. Fellows will receive a grant of $50,000 per year for up to three years and will conduct two years of stem cell and regenerative medicine research at a recognized research laboratory in Canada as well as another year of research at the Pfizer Neusentis laboratories in the United Kingdom.

The 2011 fellowship recipients that have just been announced, following an internationally publicized competition, are Dr. Corinne Hoesli from Laval University in Qubec City and Dr. Reaz Vawda from University Health Network in Toronto. Dr. Hoesli proposes to conduct research related to engineering artificial blood vessels and is speaking today at the Till and McCulloch Meetings in Montral about the program and her research strategies. The research specialty of Dr. Vawda is comparative investigations on the therapeutic repair function of mesenchymal stem cells in the treatment of spinal cord injury.

"We are very pleased to name these first recipients of the See The Potential postdoctoral fellowships in partnership with Pfizer Inc," said Dr. Verna Skanes, Chair of the Board of the Stem Cell Network. "This program is an exciting way to provide young researchers with the opportunity to develop their research efforts and their careers while building important collaborations for the future with other researchers connected to the Stem Cell Network and, internationally, through Pfizer network. This is exactly the type of collaboration with industry that is the hallmark of translational research and one that can provide benefits to all involved."

Half the program is funded by the Stem Cell Network and other half shared by Pfizer.

"This is an excellent initiative aligned with the Pfizer Neusentis' mission to develop innovative cell therapies to benefit patients through research and development, clinical and business innovation," said academic liaison, Dr. Tim Allsopp, Head of External Research for the Regenerative Medicine activities at Pfizer Neusentis Ltd. "We congratulate our winners and look forward to witnessing the results of their important research."

The second See The Potential fellowship competition is now open with an application deadline set for June 26, 2012. For more information on the competition please visit http://www.seethepotential.ca

Canada's Stem Cell Network The Stem Cell Network, established in 2001, brings together more than 100 leading scientists, clinicians, engineers, and ethicists from universities and hospitals across Canada. The Network supports cutting-edge projects that translate research discoveries into new and better treatments for millions of patients in Canada and around the world. Hosted by the University of Ottawa, the Stem Cell Network is one of Canada's Networks of Centres of Excellence funded through Industry Canada and its three granting councils. http://www.stemcellnetwork.ca

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First fellowships awarded in new Canadian stem cell and regenerative medicine research program

ScienceDaily (May 2, 2012) HIV patients treated with genetically modified T cells remain healthy up to 11 years after initial therapy, researchers from the Perelman School of Medicine at the University of Pennsylvania report in the new issue of Science Translational Medicine. The results provide a framework for the use of this type of gene therapy as a powerful weapon in the treatment of HIV, cancer, and a wide variety of other diseases.

"We have 43 patients and they are all healthy," says senior author Carl June, MD, a professor of Pathology and Laboratory Medicine at Penn Medicine. "And out of those, 41 patients show long term persistence of the modified T cells in their bodies."

Early gene therapy studies raised concern that gene transfer to cells via retroviruses might lead to leukemia in a substantial proportion of patients, due to mutations that may arise in genes when new DNA is inserted. The new long-term data, however, allay that concern in T cells, further buoying the hope generated by work June's team published in 2011 showing the eradication of tumors in patients with chronic lymphocytic leukemia using a similar strategy.

"If you have a safe way to modify cells in patients with HIV, you can potentially develop curative approaches," June says. "Patients now have to take medicine for their whole lives to keep their virus under control, but there are a number of gene therapy approaches that might be curative." A lifetime of anti-HIV drug therapy, by contrast, is expensive and can be accompanied by significant side effects.

They also note that the approach the Penn Medicine team studied may allow patients with cancers and other diseases to avoid the complications and mortality risks associated with more conventional treatments, since patients treated with the modified T cells did not require drugs to weaken their own immune systems in order for the modified cells to proliferate in their bodies after infusion, as is customary for cancer patients who receive stem cell transplants.

To demonstrate the long-term safety of genetically modified T cells, June and colleagues have followed HIV-positive patients who enrolled in three trials between 1998 and 2002. Each patient received one or more infusions of their own T cells that had been genetically modified in the laboratory using a retroviral vector. The vector encoded a chimeric antigen receptor that recognizes the HIV envelope protein and directs the modified T cell to kill any HIV-infected cells it encounters.

As is standard for any trial, the researchers carefully monitored patients for any serious adverse events immediately after infusion -- none of which were seen. Additionally, because of the earlier concerns about long-term side effects, the U.S. Food and Drug Administration also asked the team to follow the patients for up to 15 years to ensure that the modified T cells were not causing blood cancers or other late effects. Therefore, each patient underwent an exam and provided blood samples during each of the subsequent years.

Now, with more than 500 years of combined patient safety data, June and colleagues are confident that the retroviral vector system is safe for modifying T cells. By contrast, June notes, the earlier, worrying side effects were seen when viral vectors were used to modify blood stem cells. The new results show that the target cell for gene modification plays an important role in long-term safety for patients treated. "T cells appear to be a safe haven for gene modification," June says.

The multi-year blood samples also show that the gene-modified T cell population persists in the patients' blood for more than a decade. In fact, models suggest that more than half of the T cells or their progeny are still alive 16 years after infusion, which means one treatment might be able to kill off HIV-infected cells for decades. The prolonged safety data means that it might be possible to test T cell-based gene therapy for the treatment of non-life threatening diseases, like arthritis.

"Until now, we've focused on cancer and HIV-infection, but these data provide a rationale for starting to focus on other disease types," June says. "What we have demonstrated in this study and recent studies is that gene transfer to T cells can endow these cells with enhanced and novel functions. We view this as a personalized medicine platform to target disease using a patient's own cells."

Read the original:
Genetically modified T cell therapy appears to be safe, lasting in decade-long study of HIV patients

Public release date: 2-May-2012 [ | E-mail | Share ]

Contact: Holly Auer holly.auer@uphs.upenn.edu 215-200-2313 University of Pennsylvania School of Medicine

PHILADELPHIA -- HIV patients treated with genetically modified T cells remain healthy up to 11 years after initial therapy, researchers from the Perelman School of Medicine at the University of Pennsylvania report in the new issue of Science Translational Medicine. The results provide a framework for the use of this type of gene therapy as a powerful weapon in the treatment of HIV, cancer, and a wide variety of other diseases.

"We have 43 patients and they are all healthy," says senior author Carl June, MD, a professor of Pathology and Laboratory Medicine at Penn Medicine. "And out of those, 41 patients show long term persistence of the modified T cells in their bodies."

Early gene therapy studies raised concern that gene transfer to cells via retroviruses might lead to leukemia in a substantial proportion of patients, due to mutations that may arise in genes when new DNA is inserted. The new long-term data, however, allay that concern in T cells, further buoying the hope generated by work June's team published in 2011 showing the eradication of tumors in patients with chronic lymphocytic leukemia using a similar strategy.

"If you have a safe way to modify cells in patients with HIV, you can potentially develop curative approaches," June says. "Patients now have to take medicine for their whole lives to keep their virus under control, but there are a number of gene therapy approaches that might be curative." A lifetime of anti-HIV drug therapy, by contrast, is expensive and can be accompanied by significant side effects.

They also note that the approach the Penn Medicine team studied may allow patients with cancers and other diseases to avoid the complications and mortality risks associated with more conventional treatments, since patients treated with the modified T cells did not require drugs to weaken their own immune systems in order for the modified cells to proliferate in their bodies after infusion, as is customary for cancer patients who receive stem cell transplants.

To demonstrate the long-term safety of genetically modified T cells, June and colleagues have followed HIV-positive patients who enrolled in three trials between 1998 and 2002. Each patient received one or more infusions of their own T cells that had been genetically modified in the laboratory using a retroviral vector. The vector encoded a chimeric antigen receptor that recognizes the HIV envelope protein and directs the modified T cell to kill any HIV-infected cells it encounters.

As is standard for any trial, the researchers carefully monitored patients for any serious adverse events immediately after infusion -- none of which were seen. Additionally, because of the earlier concerns about long-term side effects, the U.S. Food and Drug Administration also asked the team to follow the patients for up to 15 years to ensure that the modified T cells were not causing blood cancers or other late effects. Therefore, each patient underwent an exam and provided blood samples during each of the subsequent years.

Now, with more than 500 years of combined patient safety data, June and colleagues are confident that the retroviral vector system is safe for modifying T cells. By contrast, June notes, the earlier, worrying side effects were seen when viral vectors were used to modify blood stem cells. The new results show that the target cell for gene modification plays an important role in long-term safety for patients treated. "T cells appear to be a safe haven for gene modification," June says.

View original post here:
Genetically modified T cell therapy shown to be safe, lasting in decade-long study of HIV patients

South San Francisco, CA (Marketwire) - VistaGen Therapeutics, Inc. (OTCBB:VSTA) (OTCQB:VSTA), a biotechnology company applying stem cell technology for drug rescue and cell therapy, has licensed breakthrough stem cell culture technology from the McEwen Centre for Regenerative Medicine located at the University Health Network (UHN) in Toronto, Canada.

VistaGen will be utilizing the licensed technology to develop hematopoietic precursor stem cells from human pluripotent stem cells, with the goal of developing drug screening and cell therapy applications for human blood system disorders. The breakthrough technology is included in a new United States patent application.

Hematopoietic precursor stem cells give rise to all red and white blood cells and platelets in the body. VistaGen will use the UHN invention to improve the cell culture methods used to efficiently produce hematopoietic stem cell populations.

"This technology dramatically advances our ability to produce and purify this important blood stem cell precursor for both in vitro drug screening and in vivo cell therapy applications," said H. Ralph Snodgrass, PhD, VistaGen's President and Chief Scientific Officer.

"In addition to defining new cell culture methods for our use, the technology describes the surface characteristics of stem cell-derived adult hematopoietic stem cells. Most groups study embryonic blood development from stem cells, but, for the first time, we are able to not only purify the stem cell-derived precursor of all adult hematopoietic cells, but also pinpoint the precise timing when adult blood cell differentiation takes place in these cultures," Snodgrass added. "It is our belief that these early cells will be the precursors of the ultimate adult, bone marrow-repopulating hematopoietic stem cells."

Bone marrow-derived hematopoietic stem cells are able to repopulate the blood and immune system when transplanted into patients prepared for bone marrow transplantation. These cells have important potential therapeutic applications for the restoration of healthy blood and immune systems in individuals undergoing transplantation therapies for cancer, organ grafts, HIV infections or for acquired or genetic blood and immune deficiencies.

About VistaGen Therapeutics VistaGen is a biotechnology company applying human pluripotent stem cell technology for drug rescue and cell therapy. VistaGen's drug rescue activities combine its human pluripotent stem cell technology platform, Human Clinical Trials in a Test Tube, with modern medicinal chemistry to generate new chemical variants (Drug Rescue Variants) of once-promising small-molecule drug candidates. These are drug candidates discontinued due to heart toxicity after substantial development by pharmaceutical companies, the U.S. National Institutes of Health (NIH) or university laboratories. VistaGen uses its pluripotent stem cell technology to generate early indications, or predictions, of how humans will ultimately respond to new drug candidates before they are ever tested in humans, bringing human biology to the front end of the drug development process.

Additionally, VistaGen's small molecule drug candidate, AV-101, is in Phase 1b development for treatment of neuropathic pain. Neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system, affects approximately 1.8 million people in the U.S. alone. VistaGen is also exploring opportunities to leverage its current Phase 1 clinical program to enable additional Phase 2 clinical studies of AV-101 for epilepsy, Parkinson's disease and depression. To date, VistaGen has been awarded over $8.5 million from the NIH for development of AV-101.

Visit VistaGen athttp://www.VistaGen.com, follow VistaGen athttp://www.twitter.com/VistaGenor view VistaGen's Facebook page athttp://www.facebook.com/VistaGen

Cautionary Statement Regarding Forward Looking Statements The statements in this press release that are not historical facts may constitute forward-looking statements that are based on current expectations and are subject to risks and uncertainties that could cause actual future results to differ materially from those expressed or implied by such statements. Those risks and uncertainties include, but are not limited to, risks related to regulatory approvals, the issuance and protection of patents and other intellectual property, the success of VistaGen's ongoing clinical studies, including the safety and efficacy of its drug candidate, AV-101, the failure of future drug rescue and pilot preclinical cell therapy programs related to VistaGen's stem cell technology-based Human Clinical Trial in a Test Tube platform, its ability to enter into drug rescue collaborations, risks and uncertainties relating to the availability of substantial additional capital to support VistaGen's research, development and commercialization activities, and the success of its research, development, regulatory approval, marketing and distribution plans and strategies, including those plans and strategies related to AV-101 and any drug rescue variants identified and developed by VistaGen. These and other risks and uncertainties are identified and described in more detail in VistaGen's filings with the Securities and Exchange Commission (SEC). These filings are available on the SEC's website at http://www.sec.gov. VistaGen undertakes no obligation to publicly update or revise any forward-looking statements.

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VistaGen Licenses Breakthrough Stem Cell Culture Technology To Speed Development Of Drug Screening And Cell Therapy ...

Public release date: 1-May-2012 [ | E-mail | Share ]

Contact: Charles Casey charles.casey@ucdmc.ucdavis.edu 916-734-9048 University of California - Davis Health System

UC Davis Health System researchers are a step closer to launching human clinical trials involving the use of an innovative stem cell therapy to fight the virus that causes AIDS.

In a paper published in the May issue of the Journal of Virology, the UC Davis HIV team demonstrated both the safety and efficacy of transplanting anti-HIV stem cells into mice that represent models of infected patients. The technique, which involves replacing the immune system with stem cells engineered with a triple combination of HIV-resistant genes, proved capable of replicating a normally functioning human immune system by protecting and expanding HIV-resistant immune cells. The cells thrived and self-renewed even when challenged with an HIV viral load.

"We envision this as a potential functional cure for patients infected with HIV, giving them the ability to maintain a normal immune system through genetic resistance," said lead author Joseph Anderson, an assistant adjunct professor of internal medicine and a stem cell researcher at the UC Davis Institute for Regenerative Cures. "Ideally, it would be a one-time treatment through which stem cells express HIV-resistant genes, which in turn generate an entire HIV-resistant immune system."

To establish immunity in mice whose immune systems paralleled those of patients with HIV, Anderson and his team genetically modified human blood stem cells, which are responsible for producing the various types of immune cells in the body.

Building on work that members of the team have pursued over the last decade, they developed several anti-HIV genes that were inserted into blood stem cells using standard gene-therapy techniques and viral vectors (viruses that efficiently insert the genes they carry into host cells). The resulting combination vector contained:

a human/rhesus macaque TRIM5 isoform, which disrupts HIV from uncoating in the cytoplasm a CCR5 short hairpin RNA (shRNA), which prevents certain strains of HIV from attaching to target cells a TAR decoy, which stops HIV genes from being expressed inside of the cell by soaking up a critical protein needed for HIV gene expression These engineered blood stem cells, which could be differentiated into normal and functional human immune cells, were introduced into the mice. The goal was to validate whether this experimental treatment would result in an immune system that remained functional, even in the face of an HIV infection, and would halt or slow the progression toward AIDS.

The results were successful on all counts.

"After we challenged transplanted mice with live HIV, we demonstrated that the cells with HIV-resistant genes were protected from infection and survived in the face of a viral challenge, maintaining normal human CD4 levels," said Anderson. CD4+ T-cells are a type of specialized immune cell that HIV attacks and uses to make more copies of HIV.

The rest is here:
Study using stem cell therapy shows promise in fight against HIV

ScienceDaily (May 2, 2012) UC Davis Health System researchers are a step closer to launching human clinical trials involving the use of an innovative stem cell therapy to fight the virus that causes AIDS.

In a paper published in the May issue of the Journal of Virology, the UC Davis HIV team demonstrated both the safety and efficacy of transplanting anti-HIV stem cells into mice that represent models of infected patients. The technique, which involves replacing the immune system with stem cells engineered with a triple combination of HIV-resistant genes, proved capable of replicating a normally functioning human immune system by protecting and expanding HIV-resistant immune cells. The cells thrived and self-renewed even when challenged with an HIV viral load.

"We envision this as a potential functional cure for patients infected with HIV, giving them the ability to maintain a normal immune system through genetic resistance," said lead author Joseph Anderson, an assistant adjunct professor of internal medicine and a stem cell researcher at the UC Davis Institute for Regenerative Cures. "Ideally, it would be a one-time treatment through which stem cells express HIV-resistant genes, which in turn generate an entire HIV-resistant immune system."

To establish immunity in mice whose immune systems paralleled those of patients with HIV, Anderson and his team genetically modified human blood stem cells, which are responsible for producing the various types of immune cells in the body.

Building on work that members of the team have pursued over the last decade, they developed several anti-HIV genes that were inserted into blood stem cells using standard gene-therapy techniques and viral vectors (viruses that efficiently insert the genes they carry into host cells). The resulting combination vector contained:

These engineered blood stem cells, which could be differentiated into normal and functional human immune cells, were introduced into the mice. The goal was to validate whether this experimental treatment would result in an immune system that remained functional, even in the face of an HIV infection, and would halt or slow the progression toward AIDS.

The results were successful on all counts.

"After we challenged transplanted mice with live HIV, we demonstrated that the cells with HIV-resistant genes were protected from infection and survived in the face of a viral challenge, maintaining normal human CD4 levels," said Anderson. CD4+ T-cells are a type of specialized immune cell that HIV attacks and uses to make more copies of HIV.

"We actually saw an expansion of resistant cells after the viral challenge, because other cells which were not resistant were being killed off, and only the resistant cells remained, which took over the immune system and maintained normal CD4 levels," added Anderson.

The data provided from the study confirm the safety and efficacy of this combination anti-HIV lentiviral vector in a hematopoietic stem cell gene therapy setting for HIV and validated its potential application in future human clinical trials. The team has submitted a grant application for human clinical trials and is currently seeking regulatory approval, which is necessary to move on to clinical trials.

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Stem cell therapy shows promise in fight against HIV