Inactivating Kras caused tumors to disappear, suggesting possible treatment target

Newswise — ANN ARBOR, Mich. — A mutant protein found in nearly all pancreatic cancers plays a role not only in the cancer’s development but in its continued growth, according to a new study from University of Michigan Comprehensive Cancer Center researchers. The finding suggests a possible target for developing new ways to treat this deadly disease.

Researchers have known that mutations in the Kras gene are what cause pancreatic cancer to develop. These mutations are frequently seen in common precancerous lesions, suggesting it has an early role in pancreatic cancer.

The new study, published in the February Journal of Clinical Investigation, finds that in mice, mutant Kras also keeps the tumor growing and helps precancerous tumors grow into invasive cancer. When the researchers turned off Kras, the tumors disappeared and showed no signs of recurring.

The researchers were able to manipulate Kras in a mouse model that they designed to look at Kras at various points in pancreatic cancer development. In the precancerous lesions, turning off Kras eliminated the tumors in mice and the pancreas tissue returned to normal, with no signs of the cancer returning. With invasive cancer, inactivating Kras killed off the cancer but left the pancreas with fibrous areas similar to scar tissue. Tumors did not recur.

Researchers hope this finding provides the basis for future drug development.

“Right now no drugs specifically target Kras, but there are drugs that target the cellular processes downstream of Kras. We next need to figure out which of these downstream effectors of Kras are important in pancreatic cancer,” says study author Marina Pasca di Magliano, Ph.D., assistant professor of surgery and of cell and developmental biology at the U-M Medical School.

Kras is also known to play a role in lung and colon cancer. But it is likely the biggest player in pancreatic cancer, where more than 90 percent of all tumors have mutated Kras. Pancreatic cancer is one of the most deadly types of cancer: about 4 percent of patients are alive five years after their diagnosis. The disease is often diagnosed when surgery is not an option and it tends to be resistant to available chemotherapies.

“There is a dire need for new therapies for pancreas cancer based on a better understanding of the biology of this disease. My lab is now looking at the downstream inhibitors of Kras to try to find the best target,” Pasca di Magliano says.

Note to patients: This research was based in mice and needs further testing before any possible treatments are available for clinical trials. For information about current pancreatic cancer treatments, call the U-M Cancer AnswerLine at 800-865-1125.

Pancreatic cancer statistics: 43,140 Americans will be diagnosed with pancreatic cancer this year and 36,800 will die from the disease, according to the American Cancer Society

Additional authors: Meredith A. Collins, Filip Bednar, Yaqing Zhang, Jean-christophe Brisset, Stefanie Galban, Craig J. Galban, Sabita Rakshit, and Karen S. Flannagan, all from U-M; and N. Volkan Adsay from Emory University

Funding: U-M Biological Scholar Program; National Cancer Institute; U-M Gastrointestinal Specialized Program of Research Excellence (SPORE); Pancreatic Cancer Action Network; American Association for Cancer Research; Michigan Gastrointestinal Peptide Research Center

Disclosure: None

Reference: Journal of Clinical Investigation, Vol. 122, No. 2, February 2012

Resources:
U-M Cancer AnswerLine, 800-865-1125
U-M Comprehensive Cancer Center, http://www.mcancer.org
Clinical trials at U-M, http://www.UMClinicalStudies.org/cancer

# # #

Comment/Share

Original post:
Gene Linked to Pancreatic Cancer Growth

Recommendation and review posted by Bethany Smith

WASHINGTON, D.C. — Researchers at the U.S. Department of Energy’s (DOE’s) Joint BioEnergy Institute (JBEI) recently announced a major breakthrough in engineering systems of RNA molecules through computer-assisted design.

This could lead to important improvements across a range of industries, including the development of cheaper advanced biofuels. 

Scientists will use these new “RNA machines,” to adjust genetic expression in the cells of microorganisms. 

This will enable scientists to develop new strains of Escherichia coli (E. coli) that are better able to digest switchgrass biomass and convert released sugars to form three types of transportation fuels — gasoline, diesel and jet fuels.

“This is a perfect example of how our investments in basic science innovations can pave the way for future industries and solutions to our nation’s most important challenges,” Energy Secretary Steven Chu said in a news release.

“This breakthrough at the Joint BioEnergy Institute holds enormous potential for the sustainable production of advanced biofuels and countless other valuable goods.”

A breakthrough with E. coli could make it cheaper to produce fuel from switchgrass or other non-food biomass plants to create advanced biofuels with the potential to replace gasoline. 

While the work at the JBEI remains focused on the development of advanced biofuels, its researchers believe their concepts may help other researchers to develop many other desired products, including biodegradable plastics and therapeutic drugs. 

For example, some researchers have started a project to investigate how to use the “RNA machines” to increase the safety and efficacy of medicine therapies to treat diseases, including diabetes and Parkinson’s.

Biological systems are incredibly complex, which makes it difficult to engineer systems of microorganisms that will produce desired products in predictable amounts. 

JBEI’s work, featured in the Dec. 23 issue of “Science” magazine, is the first of its kind to set up and adjust a RNA system in a predictable way. 

Specifically, researchers focused their design-driven approach on RNA sequences that can fold into complicated three dimensional shapes, called ribozymes and aptazymes. 

By using JBEI-developed computer-assisted models and simulations, researchers then created complex RNA-based control systems that are able to program a large number of genes. 

In microorganisms, “commands” that are sent into the cell will be processed by the RNA-based control systems, enabling them to help develop desired products.

One of the major goals of synthetic biology is to produce valuable chemical products from simple, inexpensive and renewable starting materials in a sustainable manner. 

Computer-assisted models and simulations like the one JBEI developed are essential for doing so. 

Up to this point, such tools for biology have been limited, and JBEI’s breakthrough in applying computer assisted design marks an important technical and conceptual achievement for this field. 

To view additional details about this research, visit http://newscenter.lbl.gov/news-releases/2011/12/22/cad-for-rna/.

The rest is here:
Genetic breakthroughs help develop cheaper biofuels: DOE

Recommendation and review posted by Bethany Smith

11-12-2011 09:20 A decade ago, scientists announced that they had produced the first draft of the human genome, the 3.6 billion letters of our genetic code. It was seen as one of the greatest scientific achievements of our age, a breakthrough that would usher in a new age of medicine. A decade later, Horizon finds out how close we are to developing the life-changing treatments that were hoped for. Horizon follows three people, each with a genetic disease, as they go behind the scenes at some of Britain's leading research labs to find out what the sequencing of the human genome has done for them - and the hope this remarkable project offers all of us.

Go here to read the rest:
Miracle Cure A Decade of the Human Genome - BBC Horizon (HD). 720p - Video

Recommendation and review posted by Bethany Smith

Public release date: 31-Jan-2012
[ | E-mail | Share ]

Contact: Katie Marquedant
kmarquedant@partners.org
617-726-0337
Massachusetts General Hospital

A report from investigators at the Massachusetts General Hospital (MGH) Cancer Center has defined the role of a recently identified gene abnormality in a deadly form of lung cancer. Tumors driven by rearrangements in the ROS1 gene represent 1 to 2 percent of non-small-cell lung cancers (NSCLC), the leading cause of cancer death in the U.S. The researchers show that ROS1-driven tumors can be treated with crizotinib, which also inhibits the growth of tumors driven by an oncogene called ALK, and describe the remarkable response of one patient to crizotinib treatment.

"ROS1 encodes a protein that is important for cell growth and survival, and deregulation of ROS1 through chromosomal rearrangement drives the growth of tumors," says Alice Shaw, MD, PhD, of the MGH Cancer Center ? co-lead author of the paper which has been published online in the Journal of Clinical Oncology. "This finding is important because we have drugs that inhibit ROS1 and could lead to the sort of dramatic clinical response we describe in this paper."

The current findings add ROS1 to the list of genes known to drive NSCLC growth when altered ? a list that includes KRAS, mutations of which account for about 25 percent of cases; EGFR, accounting for 10 to 15 percent; and ALK, rearranged in about 4 percent. Altogether, known cancer-causing genetic changes have been found in a little more than half of NSCLC tumors. Originally identified in brain tumors, ROS1 rearrangement previously had been identified in one NSCLC patient and one NSCLC cell line. The current study was designed to determine the frequency of ROS1 rearrangement in NSCLC and to define the characteristics of patients with ROS1-rearranged tumors.

The investigators screened tumor samples from more than 1,000 NSCLC patients treated at the MGH, Vanderbilt University, the University of California at Irvine, and Fudan University in Shanghai, China. ROS1 rearrangement was identified in 18 tumor samples, for a prevalence of 1.7 percent; ALK rearrangements were identified in 31 samples, with no samples showing alterations in both genes. Patients with ROS1-positive tumors tended to be younger, never to have smoked and to have a type of lung cancer called adenocarcinoma ? characteristics very similar to those of ALK-positive patients.

An earlier MGH study of an experimental ALK inhibitor had found the drug suppressed the growth of a ROS1-positive cell line in addition to ALK-positive cell lines, suggesting that ROS1-positive tumors might be sensitive to the ALK-inhibitor crizotinib. This observation led corresponding author John Iafrate, MD, PhD, and his team to develop a diagnostic test that could identify ROS1-positive tumors. Around the time that test became clinically available, a lung cancer patient whose tumor had not responded to drugs targeting EGFR mutations was referred to the MGH Cancer Center for genetic testing. His tumor was negative for ALK but later proved to harbor a ROS1 rearrangement, and he was enrolled in an extension of the crizotinib clinical trial first reported in the October 28, 2010, New England Journal of Medicine.

"When he enrolled in the trial last April, this patient was extremely sick ? with significant weight loss and very low oxygen levels ? and was barely able to walk," says Shaw. "Within a few days of starting crizotinib, he felt better; and by the time we scanned his chest at seven weeks, the tumors had essentially disappeared from his lungs." Nine months after starting crizotinib therapy, this patient continues to do well. Additional ROS1-positive patients have been enrolled in this trial at MGH, at UC Irvine and at the University of Colorado.

###

Shaw is an assistant professor of Medicine and Iafrate is an associate professor of Pathology at Harvard Medical School. Co-lead authors are Kristin Bergethon, MGH Pathology, and Sai-Hong Ignatius Ou, MD, PhD, University of California at Irvine. The study was supported by grants from the National Institutes of Health and from Pfizer, which received FDA approval for crizotinib in August 2011.

Additional co-authors are Ryohei Katayama, Eugene Mark, Julie Batten, Eunice Kwak, Jeffrey Clark, Jeffrey Engelman, and Mari Mino Kenudson, MGH Cancer Center; Christina Siwak-Tapp, University of California at Irvine; Keith D. Wilner, Pfizer; Christine Lovly, Nerina McDonald, Pierre Massion, Adriana Gonzalez, David Carbone, and William Pao, Vanderbilt University Medical Center; Pierre Massion, Nashville Veterans Affairs Medical Center; Rong Fang and Hongbin Ji, Shanghai Institutes for Biological Sciences; and Haiquan Chen, Shanghai Medical College, Fudan University.

Massachusetts General Hospital (http://www.massgeneral.org), founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH conducts the largest hospital-based research program in the United States, with an annual research budget of more than $750 million and major research centers in AIDS, cardiovascular research, cancer, computational and integrative biology, cutaneous biology, human genetics, medical imaging, neurodegenerative disorders, regenerative medicine, reproductive biology, systems biology, transplantation biology and photomedicine.

[ | E-mail | Share ]

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

More:
Massachusetts General study defines a new genetic subtype of lung cancer

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX - News) announced today that the U.S. Food and Drug Administration (FDA) has approved KALYDECOTM (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), a rare, genetic disease. KALYDECO (kuh-LYE-deh-koh) is approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Approximately 1,200 people in the United States, or 4 percent of those with CF, are believed to have this mutation. KALYDECO was granted approval in approximately three months, making it one of the fastest FDA approvals ever and marking the second approval of a new medicine from Vertex in less than a year. The company has established a financial assistance and patient support program to help get KALYDECO to eligible patients for whom it is prescribed. KALYDECO was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

Vertex is ready to support the introduction of KALYDECO and will begin shipping it to pharmacies in the United States this week. The company will host a conference call for investors and media today, January 31, 2012, at 12:15 p.m. ET to provide more information on KALYDECO availability, price and the financial assistance and patient support program.

“More than 13 years ago we set out to change the lives of people with cystic fibrosis by developing new medicines that address the underlying cause of this rare and devastating disease,” said Jeffrey Leiden, M.D., Ph.D., Vertex’s incoming President and Chief Executive Officer. “KALYDECO represents a major advance in the treatment of cystic fibrosis for people with a specific type of this disease. But our work isn’t done. With the ongoing support of doctors, patients and the Cystic Fibrosis Foundation, we’re making progress toward our ultimate goal of developing additional medicines to help many more people with cystic fibrosis.”

The approval of KALYDECO was based on data from two Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with KALYDECO experienced significant and sustained improvements in lung function as well as other disease measures, including weight gain and certain quality of life measurements, compared to those who received placebo. People who took KALYDECO also experienced significantly fewer pulmonary exacerbations, which are periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits. Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo groups. The majority of adverse events associated with KALYDECO were mild to moderate. Adverse events commonly observed in those taking KALYDECO included headache, upper respiratory tract infection (common cold), stomach pain and diarrhea.

“Advances in cystic fibrosis treatment have helped manage symptoms of the disease, however people with cystic fibrosis still have a hard time staying healthy and being active,” said Bonnie Ramsey, M.D., Director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute and principal investigator for one of the Phase 3 KALYDECO trials. “KALYDECO is a fundamental shift in the way cystic fibrosis is treated. In people with a specific genetic mutation, KALYDECO helped them breathe more easily, gain weight and generally feel better.”

“Together, we're changing the lives of people with cystic fibrosis,” said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. “We now have a medicine that treats the underlying cause of the disease in people with the G551D mutation. KALYDECO also provides us with a roadmap for exploring additional targeted approaches to treatment for all people with cystic fibrosis.”

Cystic fibrosis is a rare, life-threatening genetic disease for which there is no cure. CF is caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. CFTR proteins act as channels at the cell surface that control the flow of salt and water across the cells. When the defective CFTR protein does not work properly at the cell surface, abnormally thick, sticky mucus builds up in the lungs. The digestive tract and a number of other organs are also affected. KALYDECO, an oral medicine known as a CFTR potentiator, helps the CFTR protein function more normally once it reaches the cell surface. KALYDECO targets the abnormal CFTR protein channels and opens them to allow chloride ions to move into and out of the cell, which helps thin the mucus so it can hydrate and protect the airways, and keeps them from getting clogged and then infected.

Because KALYDECO targets a specific genetic mutation, a person’s genotype should be known before this new medicine is prescribed. Genetic testing is widely available and FDA-cleared tests are available for people with CF whose genotype is unknown. According to the 2010 Cystic Fibrosis Foundation’s Patient Registry, nearly 92 percent of people with CF have already had their CF mutations identified.

KALYDECO by itself works in a subset of people with CF, but research is ongoing to explore a similar targeted approach using a combination of medicines, including KALYDECO, to treat the most common form of the disease.

Helping People with CF Get KALYDECO

The people who work at Vertex understand that medicines can only help patients who can get them. To that end, the company offers a comprehensive financial assistance and patient support program. A specially-trained and dedicated Vertex team will provide one-on-one support to help eligible patients who are prescribed KALYDECO understand their insurance benefits and the resources that are available to help them.

For eligible patients, the program also includes the following:

Free Medicine Program: Vertex will provide KALYDECO for free to people who do not have insurance and have an annual household income of $150,000 or less; and Co-Pay Assistance Program: For patients with commercial insurance plans that cover KALYDECO and who are enrolled in the Guidance and Patient Support, or GPS, program, there will be a minimal out-of-pocket obligation after which Vertex will help cover co-pay or co-insurance costs up to 30 percent of the list price of the medicine. There is no income limit to be eligible for this program.

Some patients are not eligible for company co-pay support because they have Medicare or Medicaid coverage or live in Massachusetts. There are independent non-profit copay assistance foundations that may be able to help those patients with their out-of-pocket costs.

More information about this program is available by calling 1-877-7-KALYDECO (877-752-5933) or visiting http://www.VertexGPS.com.

About KALYDECO

KALYDECO is the first treatment to target the underlying cause of CF. The Phase 3 studies evaluated KALYDECO in people with CF ages 6 and older who had at least one copy of the G551D mutation. PERSIST, a Phase 3, open-label, 96-week extension study, is underway to evaluate the long-term safety and durability of treatment with KALYDECO. This ongoing study enrolled people who completed 48 weeks of treatment in either Phase 3 study (placebo and KALYDECO treatment groups) and met other eligibility criteria. KALYDECO will be taken as one 150-mg tablet twice daily (every 12 hours).

Vertex retains worldwide rights to develop and commercialize KALYDECO. In October 2011, Vertex submitted a marketing authorization application to the European Medicines Agency (EMA) for KALYDECO and has received agreement from the EMA for accelerated assessment in Europe. The EMA regulatory review is ongoing.

Indication and Important Safety Information

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have a certain mutation in their CF gene called the G551D mutation.

KALYDECO is not for use in people with CF due to other mutations in the CF gene. It is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CF gene.

It is not known if KALYDECO is safe and effective in children under 6 years of age.

KALYDECO should not be used with certain medicines, including the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John’s Wort.

KALYDECO can cause serious side effects. High liver enzymes in the blood have occurred in patients taking KALYDECO. Regular assessment is recommended.

The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (common cold) including sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

These are not all the possible side effects of KALYDECO. Patients should tell their healthcare providers about any side effect that bothers them or doesn't go away.

Please see full Prescribing Information for KALYDECO at http://www.KALYDECO.com.

Conference Call for Media and Investors

Vertex will host a conference call and webcast today, January 31, 2012 at 12:15 p.m. ET to provide more information about today's approval, the price of KALYDECO and Vertex's new financial assistance and patient support program. The conference call will be webcast live and a link to the webcast may be accessed from the ‘Events & Presentations' page of Vertex's website at http://www.vrtx.com.

To listen to the live call on the telephone, dial 1-877-250-8889 (United States and Canada) or 1-720-545-0001 (International). To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast.

The conference ID number for the live call and replay is 48426093.

The call will be available for replay via telephone commencing January 31, 2012 at 3:00 p.m. ET running through 5:00 p.m. ET on February 7, 2012. The replay phone number for the United States and Canada is 1-855-859-2056. The international replay number is 1-404-537-3406.

Following the live webcast, an archived version will be available on Vertex's website until 5:00 p.m. ET on February 14, 2012. Vertex is also providing a podcast MP3 file available for download on the Vertex website at http://www.vrtx.com.

About Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 38 years but the median age of death remains in the mid-20s. There are more than 1,800 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The absence of working CFTR proteins results in poor flow of salt and water across cell membranes in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

In some people, CFTR proteins are present at the cell surface but do not work properly. One type of this dysfunction is known as the G551D mutation. Approximately 4 percent of those with CF, or about 1,200 people in the United States, are believed to have this mutation. An estimated 1,000 people in Europe have the G551D mutation.

In people with the most common mutation in the CFTR gene, F508del, the CFTR protein does not reach the cell surface in normal amounts and the CFTR proteins that reach the surface do not work correctly. Nearly 90 percent of people with CF have at least one copy of the F508del mutation; approximately half of those with CF have two copies. KALYDECO is not effective in CF patients who have two copies of the F508del mutation in the CFTR gene.

Vertex’s Ongoing CF Research and Development Program

KALYDECO has been approved by the FDA for people with CF ages 6 and older who have at least one copy of the G551D mutation. Vertex is planning to begin additional studies this year to evaluate KALYDECO in children with CF as young as 2 years old and in people with CF who have the R117H mutation or gating mutations that were not evaluated in the previous Phase 3 studies.

Enrollment is ongoing in the second part of a Phase 2 clinical trial of combination regimens of KALYDECO and VX-809, a CFTR corrector, in people with the most common mutation in CF, known as F508del. In addition, the company plans to begin Phase 2 development of VX-661, a second CFTR corrector, in the first quarter of 2012.

Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)

Vertex initiated its CF research program in 1998 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. This collaboration was expanded to support the accelerated discovery and development of Vertex's CFTR modulators.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit http://www.cff.org.

About Vertex

Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.

Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis, epilepsy and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, MA, we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada. Today, Vertex has more than 2,000 employees around the world, and Science magazine named Vertex number one on its 2011 list of Top Employers in the life sciences.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding (i) Vertex being ready to support the introduction of KALYDECO and beginning to ship it to pharmacies this week; (ii) Vertex's financial assistance and patient support programs; (iii) the progress Vertex is making toward its ultimate goal of developing additional medicines to help many more people with cystic fibrosis; (iv) the roadmap provided by KALYDECO for exploring additional targeted approaches to treatment for all people with cystic fibrosis; (v) the ongoing research to explore a targeted approach using a combination of medicines, including KALYDECO, to treat the most common form of the disease and (vi) planned additional clinical trials of KALYDECO in children as young as 2 years old and people with CF who have the R117H mutation and gating mutations that were not evaluated in previous Phase 3 clinical trials. While the company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, risks related to the commercialization of KALYDECO and development of additional medicines to treat cystic fibrosis and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Photos/Multimedia Gallery Available: http://www.businesswire.com/cgi-bin/mmg.cgi?eid=50150557&lang=en

MULTIMEDIA AVAILABLE:http://www.businesswire.com/cgi-bin/mmg.cgi?eid=50150557&lang=en

View post:
FDA Approves KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis

Recommendation and review posted by Bethany Smith

Featured Article
Academic Journal
Main Category: Stem Cell Research
Also Included In: Neurology / Neuroscience;  Biology / Biochemistry
Article Date: 31 Jan 2012 - 2:00 PST

email to a friend   printer friendly   opinions  

Current Article Ratings:

Patient / Public:

5 (1 votes)

Healthcare Prof:
Bypassing the stem cell stage, researchers at the Stanford University School of Medicine in California converted mouse skin cells directly into neural precursor cells, the cells that go on to form the three main types of cell in the brain and nervous system. They write about their findings in the 30 January early online issue of the Proceedings of the National Academy of Sciences.

The findings of this and an earlier study question the idea that pluripotency (the ability to become virtually any other cell in the body, a key characteristic of stem cells) is a necessary stage in the conversion of one cell type to another.

In the earlier study, the same team transformed mouse and human skin cells directly into functional neurons. But this study is a substantial advance on the earlier one for two reasons.

First, neural precursor cells can not only differentiate into neurons, they can also become either of the two other main types of cell in the nervous system: astrocytes and oligodendrocytes.

Astrocytes are star-shaped glia cells that hold neurons in place, get nutrients to them, and digest parts of dead neurons. Oligodendrocytes make the myelin that insulates nerve fibers that connect neurons to one another and allows them to transmit signals.

And secondly, neural precursor cells are a more useful and versatile end-product for the lab, where they can be cultivated in large numbers for transplantation or drug screening.

Together, the two studies raise the possibility that embryonic stem cell research and induced pluripotency could be replaced by a more direct way of making specific cell types for treatments and research.

The problem with embryonic stem cells, although they are considered the "gold standard" in generating new types of cell, is the ethical question of where they come from, and also because they don't come from the patient's own body, the patient has to take drugs to stop their immune system rejecting the new tissue.

Induced pluripotency, where the patient's own cells are reprogrammed into stem cells, appears to overcome the ethical and immune rejection problems of embryonic stem cells, except they introduce the risk of switching on genes that cause cancer. Although this risk can be reduced by screening out unwanted pluripotent cells, it introduces a cost.

The senior author of the new study is Dr Marius Wernig, assistant professor of pathology and a member of Stanford's Institute for Stem Cell Biology and Regenerative Medicine. He told the press he and his colleagues were "thrilled" about the medical potential of their findings.

"We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons. This is important because the mouse model we used mimics that of a human genetic brain disease," said Wernig.

However, he cautioned that more work is needed before they can show a similar conversion from human skin cells is not only possible and effective, but also safe.

For the study, Wernig and colleages infected embryonic mouse skin cells (a cell line commonly used in labs) with a virus carrying three transcription factors (Brn2, Sox2 and FoxG1) known to be present at a high level in neural precursor cells. In just over three weeks, one in ten of the skin cells had started to look and act like neural precursor cells.

In the earlier study, they had used a different set of three transcription factors (Brn2, Ascl1 and Myt1l).

They confirmed the presence of neural precursor cells in two ways: in the lab and in animals (in vitro and in vivo).

In the lab, they confirmed the transformed cells were expressing the appropriate genes and had the same shape and function as naturally derived neural precursor cells.

And to confirm them in animals, they injected the new cells into the brains of newborn mice bred to lack to ability to make the myelin sheath that surrounds nerve fibers. After ten weeks, the new cells had differentiated into oligodendroytes and had begun to coat the mice's nerve fibers with myelin.

The team is now hoping to repeat their success with skin cells from adult mice and humans.

Funds from the California Institute for Regenerative Medicine, the New York Stem Cell Foundation, the Ellison Medical Foundation, the Stinehart-Reed Foundation and the National Institutes of Health helped pay for the study.

Written by Catharine Paddock PhD
Copyright: Medical News Today
Not to be reproduced without permission of Medical News Today

Visit our stem cell research section for the latest news on this subject. "Direct conversion of mouse fibroblasts to self-renewing, tripotent neural precursor cells"; Ernesto Lujan, Soham Chanda, Henrik Ahlenius, Thomas C. Südhof, and Marius Wernig; PNAS Published online ahead of print 30 January 2012; DOI:10.1073/pnas.1121003109; Link to Abstract.
Additional source: Stanford University School of Medicine Please use one of the following formats to cite this article in your essay, paper or report:

MLA

Catharine Paddock PhD. "Scientists Transform Skin Cells Direct To Brain Cells, Bypassing Stem Cell Stage." Medical News Today. MediLexicon, Intl., 31 Jan. 2012. Web.
31 Jan. 2012. <http://www.medicalnewstoday.com/articles/240943.php&gt;

APA

Please note: If no author information is provided, the source is cited instead.

Rate this article:
(Hover over the stars then click to rate) Patient / Public:
or Health Professional:

Please note that we publish your name, but we do not publish your email address. It is only used to let you know when your message is published. We do not use it for any other purpose. Please see our privacy policy for more information.

If you write about specific medications or operations, please do not name health care professionals by name.

All opinions are moderated before being included (to stop spam)

Contact Our News Editors

For any corrections of factual information, or to contact the editors please use our feedback form.

Please send any medical news or health news press releases to:

Note: Any medical information published on this website is not intended as a substitute for informed medical advice and you should not take any action before consulting with a health care professional. For more information, please read our terms and conditions.

Read the original post:
Scientists Transform Skin Cells Direct To Brain Cells, Bypassing Stem Cell Stage

Recommendation and review posted by simmons

Public release date: 27-Jan-2012
[ | E-mail | Share ]

Contact: Daniel Kane
dbkane@ucsd.edu
858-534-3262
University of California - San Diego

Stem cells derived from fat have a surprising trick up their sleeves: Encouraged to develop on a stiff surface, they undergo a remarkable transformation toward becoming mature muscle cells. The new research appears in the journal Biomaterials. The new cells remain intact and fused together even when transferred to an extremely stiff, bone-like surface, which has University of California, San Diego bioengineering professor Adam Engler and colleagues intrigued. These cells, they suggest, could hint at new therapeutic possibilities for muscular dystrophy.

In diseases like muscular dystrophy or a heart attack, "muscle begins to die and undergoes its normal wounding processes," said Engler, a bioengineering professor at the Jacobs School of Engineering at UC San Diego. "This damaged tissue is fundamentally different from a mechanical perspective" than healthy tissue.

Transplanted stem cells might be able to replace and repair diseased muscle, but up to this point the transplants haven't been very successful in muscular dystrophy patients, he noted. The cells tend to clump into hard nodules as they struggle to adapt to their new environment of thickened and damaged tissue.

Engler, postdoctoral scholar Yu Suk Choi and the rest of the team think their fat-derived stem cells might have a better chance for this kind of therapy, since the cells seem to thrive on a stiff and unyielding surface that mimics the damaged tissue found in people with MD.

In their study in the journal Biomaterials, the researchers compared the development of bone marrow stem cells and fat-derived stem cells grown on surfaces of varying stiffness, ranging from the softness of brain tissue to the hardness of bone.

Cells from the fat lineage were 40 to 50 times better than their bone marrow counterparts at displaying the proper proteins involved in becoming muscle. These proteins are also more likely to "turn on" in the correct sequence in the fat-derived cells, Engler said.

Subtle differences in how these two types of cells interact with their environment are critical to their development, the scientists suggest. The fat-derived cells seem to sense their "niche" on the surfaces more completely and quickly than marrow-derived cells. "They are actively feeling their environment soon, which allows them to interpret the signals from the interaction of cell and environment that guide development," Choi explained.

Perhaps most surprisingly, muscle cells grown from the fat stem cells fused together, forming myotubes to a degree never previously observed. Myotubes are a critical step in muscle development, and it's a step forward that Engler and colleagues hadn't seen before in the lab.

The fused cells stayed fused when they were transferred to a very stiff surface. "These programmed cells are mature enough so that they don't respond the environmental cues" in the new environment that might cause them to split apart, Engler says.

Engler and colleagues will now test how these new fused cells perform in mice with a version of muscular dystrophy. The cells survive in an environment of stiff tissue, but Engler cautions that there are other aspects of diseased tissue such as its shape and chemical composition to consider. "From the perspective of translating this into a clinically viable therapy, we want to know what components of the environment provide the most important cues for these cells," he said.

###

Co-authors for the Biomaterials study "Mechanical derivation of functional myotubes from adipose-derived stem cells" include Ludovic G. Vincent and Andrew R. Lee in the Department of Bioengineering at the UC San Diego Jacobs School of Engineering, and Marek K. Dobke from the Division of Plastic Surgery, UC San Diego School of Medicine. The research was funded by the Human Frontier Science Program and the National Institutes of Health Common Fund.

[ | E-mail | Share ]

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

Link:
Need muscle for a tough spot? Turn to fat stem cells, UC San Diego researchers say

Recommendation and review posted by Bethany Smith

11-08-2011 09:02 Dr. Jennifer Ashton discusses an experimental gene therapy that used modified T-cells to attack tumor cells.

View post:
Promising gene therapy treats Leukemia - Video

Recommendation and review posted by Bethany Smith

SOURCE: Cytori Therapeutics, Inc.

SAN DIEGO, CA--(Marketwire - Jan 30, 2012) - Cytori Therapeutics (NASDAQ: CYTX) has received an Investigational Device Exemption (IDE) approval from the U.S. FDA to begin the ATHENA trial. ATHENA will investigate the use of the Celution® System, an innovative medical device to prepare adipose-derived stem and regenerative cells (ADRCs) to treat a form of coronary heart disease, chronic myocardial ischemia (CMI). The IDE application was originally submitted to the FDA in December 2011.

ATHENA is a multi-center, randomized, double blind, placebo controlled, pilot trial to investigate the use of autologous, clinical-grade ADRCs, processed at the point-of-care with Cytori's proprietary Celution® System. The trial will enroll up to 45 patients with no-option CMI who have limited therapeutic options. It will evaluate a variety of clinical and functional outcomes, including safety, peak oxygen consumption (mVO2), and clinical outcomes at 12-months.

"Following our pre-IDE meeting with the FDA, we received constructive guidance and implemented the Agency's recommendations, ultimately resulting in rapid approval to initiate the ATHENA trial," said Christopher J. Calhoun, chief executive officer for Cytori. "We look forward to working with the FDA on further defining our clinical strategy in the U.S."

Previously, Cytori reported six and 18-month trial data from PRECISE, a European clinical trial for this same indication showing improvement in mVO2. In Europe, Cytori has applied to expand its Celution® System CE Mark to include no-option CMI claims based on data from the PRECISE trial. Cytori is also enrolling ADVANCE, a European pivotal trial investigating the Celution® System for acute myocardial infarction (heart attacks).

In the U.S., it is estimated that 120,000 to 250,000 patients are diagnosed each year with chronic myocardial ischemia, a subset of the approximate 5.8 million patients who currently have some form of heart failure. CMI patients typically have undergone multiple revascularization procedures that have not improved their condition and are at a stage where they have few therapeutic options remaining.

About Cytori
Cytori is a leader in providing patients and physicians around the world with medical technologies that harness the potential of adult regenerative cells from adipose tissue. The Celution® System family of medical devices and instruments is being sold into the European and Asian cosmetic and reconstructive surgery markets and available in the United States only for use as an investigational device under Cytori's FDA approved IDE. Our StemSource® product line is sold globally for cell banking and research applications. Our PureGraft® products are available in North America and Europe for fat grafting procedures. http://www.cytori.com

Cautionary Statement Regarding Forward-Looking Statements
This press release includes forward-looking statements regarding events, trends and business prospects, which may affect our future operating results and financial position, such as the successful initiation of a clinical trial of the Company's Celution® System for chronic myocardial ischemia, our efforts to expand our CE Mark. Such statements are subject to risks and uncertainties that could cause our actual results and financial position to differ materially. Some of these risks include clinical and regulatory uncertainties, such as those associated with the ATHENA clinical trial, including risks in the collection and results of clinical data, final clinical outcomes, dependence on third party performance, and other risks and uncertainties described under the "Risk Factors" in Cytori's Securities and Exchange Commission Filings. We assume no responsibility to update or revise any forward-looking statements to reflect events, trends or circumstances after the date they are made.

Go here to see the original:
Cytori Receives Approval From FDA to Initiate U.S. Cardiac Cell Therapy Trial; Investigational Device Exemption (IDE ...

Recommendation and review posted by Bethany Smith

RANCHO CORDOVA, Calif., Jan. 30, 2012 /PRNewswire/ -- ThermoGenesis Corp. (NASDAQ: KOOL - News), a leading supplier of innovative products and services that process and store adult stem cells, today announced that the Company has implemented a number of changes in corporate management responsibilities to maximize the Company's progress toward its strategic goals.

Matthew Plavan, currently Chief Financial Officer and Executive Vice President, Business Development, will become Chief Executive Officer and a member of the Board of Directors, while retaining his position as the Company's Chief Financial Officer. Plavan has served as Chief Financial Officer since 2005 and served as Chief Operating Officer from 2008-2010.

Plavan replaces J. Melville Engle, who has retired from his position as Chairman and Chief Executive Officer.  Engle joined the Company as Chief Executive Officer in 2009 and was named Chairman of the Board in 2010. "ThermoGenesis has made important strides during Mel's tenure, as he was instrumental in building the ThermoGenesis management team and in the expansion of the Company's distributor network. We appreciate his contributions to the Company," said Patrick McEnany, a member of the board of directors.

"Given the uncertain duration of today's challenging global economy, we chose to reorganize the Company.  The board believed changes were necessary if the Company was going to achieve its short-term objectives and be positioned for long-term growth. We believe Matt's experience, knowledge of our market and proven track record at ThermoGenesis makes him the most qualified person to lead the Company," McEnany added.

"I appreciate the board's confidence in me and believe our streamlined management is a strong and dedicated group of individuals that will drive the Company to success," Plavan stated.  "Our current organization was staffed to support a faster entry of our products into new markets than we have achieved as of today, including China and India. The changes made today recalibrate the Company's resources to our current revenues and the cadence of new market opportunities, while maintaining the strong support our growing customer base has come to expect. We believe we are now optimally positioned to grow the business and maximize shareholder value, even in these turbulent economic times," Plavan added.

The Company indicated it has also eliminated eight additional positions. The Company will provide additional details on its new operating structure and objectives during its second quarter fiscal 2012 conference call on Thursday, February 9th.

The Company said it expects to record one-time expenses of approximately $500,000 related to the reorganization announced today in the third quarter of fiscal 2012.  The restructured operations should result in an annualized expense reduction of approximately $2 million.

About ThermoGenesis Corp.

ThermoGenesis Corp. (www.thermogenesis.com) is a leader in developing and manufacturing automated blood processing systems and disposable products that enable the manufacture, preservation and delivery of cell and tissue therapy products. These include:

The BioArchive® System, an automated cryogenic device, used by cord blood stem cell banks in more than 30 countries for cryopreserving and archiving cord blood stem cell units for transplant. AXP® AutoXpress® Platform (AXP), a proprietary family of automated devices that includes the AXP and the MXP® MarrowXpress® and companion sterile blood processing disposables for harvesting stem cells in closed systems. The AXP device is used for the processing of cord blood. The MXP is used for the preparation of cell concentrates, including stem cells, from bone marrow aspirates in the laboratory setting. The Res-Q® 60 BMC/PRP (Res-Q), a point-of-care system designed for the preparation of cell concentrates, including stem cells, from bone marrow aspirates and whole blood for platelet rich plasma (PRP). The CryoSeal® FS System, an automated device and companion sterile blood processing disposable, used to prepare fibrin sealants from plasma in about an hour. The CryoSeal FS System is approved in the U.S. for liver resection surgeries. The CryoSeal FS System has received the CE-Mark which allows sales of the product throughout the European community.

This press release contains forward-looking statements.  These statements involve risks and uncertainties that could cause actual outcomes to differ materially from those contemplated by the forward-looking statements. Several factors including timing of FDA and foreign regulatory approvals, changes in customer forecasts, our failure to meet customers' purchase order and quality requirements, supply shortages, production delays, changes in the markets for customers' products, introduction timing and acceptance of our new products scheduled for fiscal year 2012, and introduction of competitive products and other factors beyond our control could result in a materially different revenue outcome and/or in our failure to achieve the revenue levels we expect for fiscal 2012.  A more complete description of these and other risks that could cause actual events to differ from the outcomes predicted by our forward-looking statements is set forth under the caption "Risk Factors" in our annual report on Form 10-K and other reports we file with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.

ThermoGenesis Corp.
Web site: http://www.thermogenesis.com
Contact: Investor Relations
+1-916-858-5107, or
ir@thermogenesis.com

Link:
ThermoGenesis Announces Management Transition in Conjunction With Tactical Realignment of Company

Recommendation and review posted by Bethany Smith

Sangeeta Bhatia, MIT professor of health sciences and technology and electrical engineering and computer science

Researchers at MIT and their colleagues said they have devised a way to produce liver-like cells from stem cells, a key step in studying why people respond differently to Hepatitis C.        
     
An infectious disease that can cause inflammation and organ failure, Hepatitis C has different effects on different people, but no one is sure why, the researchers said in a press release from MIT. Some people are very susceptible to the infection, while others are resistant.

The researchers said that by studying liver cells from different people in the lab, they may determine how genetic differences produce these varying responses. However, liver cells are hard to get and very difficult to grow in a lab dish because they tend to lose their normal structure and function when removed from the body.

The researchers, from MIT, Rockefeller University and the Medical College of Wisconsin, have come up with a way to produce liver-like cells from induced pluripotent stem cells (iPSCs), which are made from body tissues rather than embryos. Those liver-like cells can then be infected with Hepatitis C and help scientists study the varying responses to the infection.

The scientists claim this is the first time an infection has been made in cells derived from iPSCs. Their new technique is described in the Jan. 30 issue of the Proceedings of the National Academy of Sciences. The development, they said, may also eventually enable personalized medicine, in which doctors could test the effect of different drugs on tissues derived from the patient being treated and then customize therapy for that patient.

The new study is a collaboration between Sangeeta Bhatia, professor of health sciences and technology and electrical engineering and computer science at MIT; Charles Rice, professor of virology at Rockefeller; and Stephen Duncan, professor of human and molecular genetics at the Medical College of Wisconsin.

The iPSCs are derived from normal body cells, often skin cells. By turning on certain genes in those cells, the scientists can revert them to an immature state that is identical to embryonic stem cells, which can turn into any cell type. Once the cells become pluripotent, they can be directed to become liver-like cells by turning on genes that control liver development.

The researchers’ goal is to take cells from patients who have unusual reactions to hepatitis C infection, transform them into liver cells and study their genetics to see why people respond as they do. “Hepatitis C virus causes an unusually robust infection in some people, while others are very good at clearing it. It’s not yet known why those differences exist,” Bhatia said in a statement.

Bhatia is a 2009 Mass High Tech Women to Watch honoree.
 

More:
Stem cells may shed light on hepatitis, MIT researchers find

Recommendation and review posted by Bethany Smith

Sixteen years after Dolly the sheep was cloned in Edinburgh, scientists in Scotland have made another startling medical breakthrough.

Researchers at Edinburgh's Centre for Regenerative Medicine have created brain tissue from patients suffering mental illnesses such as schizophrenia and depression.

"A patient's neurones can tell us a great deal about the psychological conditions that affect them, but you cannot stick a needle in someone's brain and take out its cells," the center's director, Professor Charles ffrench-Constant, told the Guardian.

"However, we have found a way round that. We can take a skin sample, make stem cells from it and then direct these stem cells to grow into brain cells. Essentially, we are turning a person's skin cells into brain."

The scientists hope that studying these manufactured brain cells will reveal clues to the conditions of patients with mental illnesses—a task that had been challenging in the past.

"It is very difficult to get primary tissue to study until after a patient has died," said the Royal Edinburgh Hospital's Professor Andrew McIntosh, who is collaborating with the center on the project.

"Even then, that tissue is affected by whatever killed them and by the impact of the medication they had been taking for their condition, possibly for several decades. So having access to living brain cells is a significant development for the development of drugs for these conditions," McIntosh said.

If successful, the same methods could be used for other organs, including the liver and heart.

Click here to read more. 

Read the original:
Scientists use skin samples to create human brain cells

Recommendation and review posted by Bethany Smith

30 January 2012 Last updated at 19:13 ET By James Gallagher Health and science reporter, BBC News

Skin cells have been converted directly into cells which develop into the main components of the brain, by researchers studying mice in California.

The experiment, reported in Proceedings of the National Academy of Sciences, skipped the middle "stem cell" stage in the process.

The researchers said they were "thrilled" at the potential medical uses.

Far more tests are needed before the technique could be used on human skin.

Stem cells, which can become any other specialist type of cell from brain to bone, are thought to have huge promise in a range of treatments. Many trials are taking place, such as in stroke patients or specific forms of blindness.

One of the big questions for the field is where to get the cells from. There are ethical concerns around embryonic stem cells and patients would need to take immunosuppressant drugs as any stem cell tissue would not match their own.

An alternative method has been to take skin cells and reprogram them into "induced" stem cells. These could be made from a patient's own cells and then turned into the cell type required, however, the process results in cancer-causing genes being activated.

Continue reading the main story “Start Quote

We are thrilled about the prospects for potential medical use of these cells”

End Quote Prof Marius Wernig Stanford University School of Medicine Direct approach

The research group, at the Stanford University School of Medicine in California, is looking at another option - converting a person's own skin cells into specialist cells, without creating "induced" stem cells. It has already transformed skin cells directly into neurons.

This study created "neural precursor" cells, which can develop into three types of brain cell: neurons, astrocytes and oligodendrocytes.

These precursor cells have the advantage that, once created, they can be grown in a laboratory into very large numbers. This could be critical if the cells were to be used in any therapy.

Brain cells and skin cells contain the same genetic information, however, the genetic code is interpreted differently in each. This is controlled by "transcription factors".

The scientists used a virus to infect skin cells with three transcription factors known to be at high levels in neural precursor cells.

After three weeks about one in 10 of the cells became neural precursor cells.

Lead researcher Prof Marius Wernig said: "We are thrilled about the prospects for potential medical use of these cells.

"We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons.

"More work needs to be done to generate similar cells from human skin cells and assess their safety and efficacy."

Dr Deepak Srivastava, who has researched converting cells into heart muscle, said the study: "Opens the door to consider new ways to regenerate damaged neurons using cells surrounding the area of injury."

Go here to see the original:
Skin transformed into brain cells

Recommendation and review posted by Bethany Smith

The multiple successes of the direct conversion method could refute the idea that pluripotency (a term that describes the ability of stem cells to become nearly any cell in the body) is necessary for a cell to transform from one cell type to another. Together, the results raise the possibility that embryonic stem cell research and another technique called "induced pluripotency" could be supplanted by a more direct way of generating specific types of cells for therapy or research.

This new study, which will be published online Jan. 30 in the Proceedings of the National Academy of Sciences, is a substantial advance over the previous paper in that it transforms the skin cells into neural precursor cells, as opposed to neurons. While neural precursor cells can differentiate into neurons, they can also become the two other main cell types in the nervous system: astrocytes and oligodendrocytes. In addition to their greater versatility, the newly derived neural precursor cells offer another advantage over neurons because they can be cultivated to large numbers in the laboratory — a feature critical for their long-term usefulness in transplantation or drug screening.

In the study, the switch from skin to neural precursor cells occurred with high efficiency over a period of about three weeks after the addition of just three transcription factors. (In the previous study, a different combination of three transcription factors was used to generate mature neurons.) The finding implies that it may one day be possible to generate a variety of neural-system cells for transplantation that would perfectly match a human patient.

"We are thrilled about the prospects for potential medical use of these cells," said Marius Wernig, MD, assistant professor of pathology and a member of Stanford's Institute for Stem Cell Biology and Regenerative Medicine. "We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons. This is important because the mouse model we used mimics that of a human genetic brain disease. However, more work needs to be done to generate similar cells from human skin cells and assess their safety and efficacy."

Wernig is the senior author of the research. Graduate student Ernesto Lujan is the first author.

While much research has been devoted to harnessing the pluripotency of embryonic stem cells, taking those cells from an embryo and then implanting them in a patient could prove difficult because they would not match genetically. An alternative technique involves a concept called induced pluripotency, first described in 2006. In this approach, transcription factors are added to specialized cells like those found in skin to first drive them back along the developmental timeline to an undifferentiated stem-cell-like state. These "iPS cells" are then grown under a variety of conditions to induce them to re-specialize into many different cell types.

Scientists had thought that it was necessary for a cell to first enter an induced pluripotent state or for researchers to start with an embryonic stem cell, which is pluripotent by nature, before it could go on to become a new cell type. However, research from Wernig's laboratory in early 2010 showed that it was possible to directly convert one "adult" cell type to another with the application of specialized transcription factors, a process known as transdifferentiation.

Wernig and his colleagues first converted skin cells from an adult mouse to functional neurons (which they termed induced neuronal, or iN, cells), and then replicated the feat with human cells. In 2011 they showed that they could also directly convert liver cells into iN cells.

"Dr. Wernig's demonstration that fibroblasts can be converted into functional nerve cells opens the door to consider new ways to regenerate damaged neurons using cells surrounding the area of injury," said pediatric cardiologist Deepak Srivastava, MD, who was not involved in these studies. "It also suggests that we may be able to transdifferentiate cells into other cell types." Srivastava is the director of cardiovascular research at the Gladstone Institutes at the University of California-San Francisco. In 2010, Srivastava transdifferentiated mouse heart fibroblasts into beating heart muscle cells.

"Direct conversion has a number of advantages," said Lujan. "It occurs with relatively high efficiency and it generates a fairly homogenous population of cells. In contrast, cells derived from iPS cells must be carefully screened to eliminate any remaining pluripotent cells or cells that can differentiate into different lineages." Pluripotent cells can cause cancers when transplanted into animals or humans.

The lab's previous success converting skin cells into neurons spurred Wernig and Lujan to see if they could also generate the more-versatile neural precursor cells, or NPCs. To do so, they infected embryonic mouse skin cells — a commonly used laboratory cell line — with a virus encoding 11 transcription factors known to be expressed at high levels in NPCs. A little more than three weeks later, they saw that about 10 percent of the cells had begun to look and act like NPCs.

Repeated experiments allowed them to winnow the original panel of 11 transcription factors to just three: Brn2, Sox2 and FoxG1. (In contrast, the conversion of skin cells directly to functional neurons requires the transcription factors Brn2, Ascl1 and Myt1l.) Skin cells expressing these three transcription factors became neural precursor cells that were able to differentiate into not just neurons and astrocytes, but also oligodendrocytes, which make the myelin that insulates nerve fibers and allows them to transmit signals. The scientists dubbed the newly converted population "induced neural precursor cells," or iNPCs.

In addition to confirming that the astrocytes, neurons and oligodendrocytes were expressing the appropriate genes and that they resembled their naturally derived peers in both shape and function when grown in the laboratory, the researchers wanted to know how the iNPCs would react when transplanted into an animal. They injected them into the brains of newborn laboratory mice bred to lack the ability to myelinate neurons. After 10 weeks, Lujan found that the cells had differentiated into oligodendroytes and had begun to coat the animals' neurons with myelin.

"Not only do these cells appear functional in the laboratory, they also seem to be able to integrate appropriately in an in vivo animal model," said Lujan.

The scientists are now working to replicate the work with skin cells from adult mice and humans, but Lujan emphasized that much more research is needed before any human transplantation experiments could be conducted. In the meantime, however, the ability to quickly and efficiently generate neural precursor cells that can be grown in the laboratory to mass quantities and maintained over time will be valuable in disease and drug-targeting studies.

"In addition to direct therapeutic application, these cells may be very useful to study human diseases in a laboratory dish or even following transplantation into a developing rodent brain," said Wernig.

Provided by Stanford University Medical Center (news : web)

Read more:
Researchers turn skin cells into neural precusors, bypassing stem-cell stage

Recommendation and review posted by Bethany Smith

23-09-2011 04:14 Further information, videos, are available in topic addressed play lists and complementary video responses. Thanks to the channel: http://www.youtube.com for the Tip. "Genetically modified organisms (GMOs) in your food may make you sick. Studies link GMOs with toxins, allergies, infertility, infant mortality, immune dysfunction, stunted growth, accelerated aging, and death. Whistleblowers were fired, threatened, and gagged. Warnings by FDA scientists were ignored. Expert Jeffrey M. Smith, author of the #1 GMO bestseller Seeds of Deception, and Genetic Roulette, presents SHOCKING evidence why these gene-spliced crops may lead to health and environmental catastrophes. Learn how to protect yourself and discover the Campaign for Healthier Eating in America—a brilliant plan to quickly end the genetic engineering of our food supply." My other three research platforms: http://www.youtube.com http://www.youtube.com http://www.youtube.com

Read the original:
Jeffrey Smith Dangerous Genetically modified Foods Genetic Roulette Monsanto - Video

Recommendation and review posted by Bethany Smith

By ALLISON STROUSE
astrouse@MorningJournal.com
@AllisonStrouse

MORNING JOURNAL/JIM BOBEL Lorain County Community College students will travel to Vancouver, Canada, in February to present the two projects relating to HIV. Pictured from left to right in front are Alex Fulton, Aryel Clark, Alexis Shawver, Victoria Soewarna and Elyria Catholic High School Mark A. Jaworski. In back are Austyn Lilly, Eric McCallister, Megan Sheldon, Harry Kestler, Jacqueline Makowski and Connor Anderson.

ELYRIA — A group of nine students sat in a second-floor classroom at Lorain County Community College, discussing a deletion mutation gene that they believe could keep someone from contracting HIV and why HIV wasn’t passed on to a Florida girl at birth from her infected mother.

Those are heady topics for anyone, but when eight of the students are high-school aged, its impressive.

Harry Kestler, professor of microbiology at LCCC, and the eight Early College students and an adult student, are members of a research group at the college. Five of them are preparing to travel to Vancouver, Canada, in February to present the two projects and compete with other college students.

In a little less than a month Megan, along with Kestler; Alex Fulton, 16; Victoria Soewarna, 16; Connor Anderson, 16; and Eric McCallister, 32, will be in Vancouver at the American Association for the Advancement of Science annual meeting.

Other students in the research group are Alexis Shawver, 15; Jacqueline Makowski, 15; Aryel Clarke, 16; and Austyn Lilly, 16.

The second project looks at what kept a young girl from Florida from contracting HIV, when her mother gave it to both her younger and older siblings during birth.

“We figured out that all of the children did get the virus from their mother, so it became puzzling as to why she didn’t get infected,” Aryel Clarke, 16, said.

The group explains their projects with ease, only stopping every once in a while to make sure they are explaining it correctly.

The projects are a part of a “research fellowship” that was created after some of the early college students showed interest, according to Kestler.

“Some of them refused to leave,” Kestler recalled of a day in which they shadowed him. Continued...

The program began with more students and over time the numbers dwindled to the current nine students. Of those nine, only one, McCallister, is a “regular” college student.

“The first time I met any of them (the early college students) ... I was blown away by what they knew, I mean for high school kids,” McCallister said. “I’m really impressed by all of them. They know their stuff.”

He has seen other college students come and go from the group, lasting a day, a week, or maybe a month.

“These core group of high school kids have more determination and dedication than any of the college kids that came through here,” he said.

In February, their dedication will be put to test against students from colleges such as Harvard, Duke, and other renowned colleges, according to Kestler.

“They will be competing at the college level and not competing at the high school level,” Kestler said. “We will see how we do up against the big league players.”

The Early College students chosen for the research group are picked following their freshman year, allowing them to participate in the research group for three years, if they chose.

“At the end of the freshman years, we selected students to be in the program,” he said.

“We have some students drop out because they realize it is just not their thing,” Kestler said. “That’s OK. This is something extra.”

The ones with the program now, he expects to be back next year. Continued...

“These are students that are dedicated and they are here for the long haul,” Kestler said.

By being there, they have the chance to partake in university level research, which is one of the reasons for the program, according to Kestler.

“We have a lab here,” he said pointing out that it is not specifically set up for them, but the whole college.

“It’s really like a once in a lifetime opportunity,” Megan said. “I think I can speak for all of us and say that we are really grateful to get to do this.”

This program has helped her realize what she wants to do once she graduates.

“I know I want to do some type of research,” she said. “I can’t picture myself not being in a bio-tech lab at least once a week.”

“This is a dedication you have to have,” Megan said. “You have to be able to want this.”

Link:
LCCC students studying gene mutation in HIV fight

Recommendation and review posted by Bethany Smith

Public release date: 30-Jan-2012
[ | E-mail | Share ]

Contact: Emily Benson
SRC@faseb.org
301-634-7010
Federation of American Societies for Experimental Biology

Bethesda, MD ? The Federation of American Societies for Experimental Biology (FASEB) announces the opening of registration for the Science Research Conference (SRC): Post-transcriptional Control of Gene Expression: Mechanisms of mRNA Decay.

This conference will take place June 24-29, 2012 in Steamboat Springs, Colorado. The goal of the conference is to improve our current understanding of the fundamental mechanisms by which post-transcriptional control of RNA stability and translation regulates gene expression in normal and disease states. The program will consist of sessions that will broadly address cutting-edge issues including 5' and 3' end control of mRNA decay, subcellular localization of mRNA translation and decay, and RNA-binding proteins and mRNA fate. Small poster sessions will also enable all participants to contribute to, and learn about, these topics. There is also free time in the program for informal discussions among established and junior principal investigators, post-doctoral trainees and graduate students from the U.S. and abroad. This meeting will bring together sets of researchers who, despite having converging interests, have infrequent opportunities to meet as a group. A particular attraction is to bring together researchers focused on prokaryotic and eukaryotic features of post-transcriptional control to exchange information and stimulate new ideas. The resulting discussions and cross-fertilization will help define critical areas to propel this field forward.

###

Since 1982, FASEB SRC has offered a continuing series of inter-disciplinary exchanges that are recognized as a valuable complement to the highly successful society meetings. Divided into small groups, scientists from around the world meet intimately and without distractions to explore new approaches to those research areas undergoing rapid scientific changes.

In recent years, the SRC series has expanded into non-summer months. To better enhance the SRC series and allow for future expansion of conferences, FASEB's Office of Scientific Meetings and Conferences recently changed the SRC name from Summer Research Conferences to Science Research Conferences.

FASEB SRC has announced a total of 36 SRCs in 2012, spanning from June through October. To register for an SRC, view preliminary programs, or find a listing of all our 2012 SRCs, please visit http://www.faseb.org/SRC.

Additionally, in efforts to continue expanding the SRC series, potential organizers are encouraged to contact SRC staff at SRC@faseb.org. Proposal guidelines can be found by clicking "Submit a Proposal" on our website at http://www.faseb.org/SRC.

FASEB is composed of 26 societies with more than 100,000 members, making it the largest coalition of biomedical research associations in the United States. Celebrating 100 Years of Advancing the Life Sciences in 2012, FASEB is rededicating its efforts to advance health and well-being by promoting progress and education in biological and biomedical sciences through service to our member societies and collaborative advocacy.

[ | E-mail | Share ]

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

View original post here:
FASEB SRC announces conference registration open for: Post-transcriptional Control of Gene Expression: Mechanisms of ...

Recommendation and review posted by Bethany Smith

MONDAY, Jan. 30 (HealthDay News) -- Researchers studying the link between diabetes and a hormone that affects your so-called "body clock" have identified a genetic mutation in the receptor for the hormone, melatonin, that may to boost the risk of the disease.

The finding could help improve assessment of a person's diabetes risk and could also lead to the development of personalized treatments, according to the study published in the Jan. 29 online edition of the journal Nature Genetics.

The research team from Imperial College London found that people who have rare genetic mutations in the receptor for melatonin have a greatly increased risk for type 2 diabetes.

Melatonin controls the body's sleep-wake cycle. A previous study found that people with common variations in the gene for the melatonin receptor MT2 have a slightly increased risk for type 2 diabetes.

This new study discovered that having any of four rare mutations of the MT2 is associated with a six times increased risk of developing type 2 diabetes.

Melatonin controls the release of insulin, which regulates blood sugar levels. Mutations in the MT2 gene may disrupt the connection between the body clock and insulin release, resulting in abnormal control of blood sugar, the researchers explained.

For their study, the investigators examined the MT2 gene in more than 7,000 people. They identified 40 variants associated with type 2 diabetes, four of which are very rare and make the receptor incapable of responding to melatonin. The effect of these four variants was then confirmed in an additional group of nearly 12,000 people.

"Blood sugar control is one of the many processes regulated by the body's biological clock. This study adds to our understanding of how the gene that carries the blueprint for a key component in the clock can influence people's risk of diabetes," study leader Philippe Froguel, from the School of Public Health, said in an Imperial College London news release.

"We found very rare variants of the MT2 gene that have a much larger effect than more common variants discovered before. Although each mutation is rare, they are common in the sense that everyone has a lot of very rare mutations in their DNA. Cataloging these mutations will enable us to much more accurately assess a person's risk of disease based on their genetics," Froguel added.

While the study found a link between the mutation and diabetes risk, it did not find a cause-and-effect relationship.

More information

The American Diabetes Association offers an overview of diabetes prevention.

Read this article:
Gene Study Sheds Light on Body Clock's Link to Diabetes

Recommendation and review posted by Bethany Smith

Bill Gates has a terse response to criticism that the high-tech solutions he advocates for world hunger are too expensive or bad for the environment:  Countries can embrace modern seed technology and genetic modification or their citizens will starve.

When he was in high school in the 1960s, people worried there wouldn’t be enough food to feed the world, Gates recalled in his fourth annual letter, which was published online on January 24 and reported on by the AP in the Huffington Post. But the “green revolution,” which transformed agriculture with high-yield crop varieties and other innovations, warded off famine.

Gates is among those who believe another, similar revolution is needed now. The Bill & Melinda Gates Foundation has spent about $2 billion in the past five years to fight poverty and hunger in Africa and Asia, and much of that money has gone toward improving agricultural productivity.

Gates doesn’t apologize for his endorsement of modern agriculture or sidestep criticism of genetic modification. He told the Associated Press that he finds it ironic that most people who oppose genetic engineering in plant breeding live in rich nations that he believes are responsible for global climate change that will lead to more starvation and malnutrition for the poor.

In his 24-page letter, the Microsoft Corp. chairman lamented that more money isn’t spent on agriculture research and noted that of the $3 billion spent each year on work on the seven most important crops, only 10 percent focuses on problems in poor countries.

“Given the central role that food plays in human welfare and national stability, it is shocking – not to mention short-sighted and potentially dangerous – how little money is spent on agricultural research,” he wrote in his letter, calling for wealthier nations to step up.

Go here to read the rest:
Bill Gates: Embrace Genetic Modification or Starve

Recommendation and review posted by Bethany Smith

22-01-2012 23:39 Jan. 23 (Bloomberg) -- Dana Nieder, mother of three-year-old Maya Nieder, talks with Bloomberg's John Lauerman about her daughter's undiagnosed genetic disorder and her struggles with medical and insurance bureaucracies for advanced testing. (Source: Bloomberg)

Original post:
Mother Seeks Answers to Daughter's Genetic Illness - Video

Recommendation and review posted by Bethany Smith

In this June 6, 2011 file photo, former Pennsylvania Sen. Rick Santorum holds his daughter Bella before announcing he is entering the Republican presidential race, on the steps of the Somerset County Courthouse in Somerset, Pa. Bella has the genetic disorder Trisomy 18, and was hospitalized over the weekend with pneumonia.

(Credit: AP) (CBS) Bella Santorum, the 3-year-old daughter of Republican presidential candidate Rick Santorum, was hospitalized over the weekend with pneumonia and complications from the genetic disorder, Trisomy 18.

Also known as Edwards syndrome, Trisomy 18 occurs when a person is born with three copies of the 18th chromosome, as opposed to two. That extra chromosome interferes with typical childhood development, causing children to be born with clenched hands, crossed legs, feet with rounded bottoms, a small head and jaw, and intellectual disabilities. The disorder can also cause serious heart and kidney problems. Trisomy 18 occurs in about one out of every 3,000 births. It is three times more common in girls than boys, according to the National Institutes of Health.

Unlike Down syndrome, which is caused by an extra chromosome 21, the issues caused by Trisomy 18 are associated with more life-threatening medical complications and 50 percent of babies with Trisomy 18 who are carried to term are stillborn, according to the Trisomy 18 Foundation.

"When a child is born in this situation, they very rarely make it past the first week, because one or two problems can be overwhelming and it just kind of piles up," Dr. Brian McDonough, clinical professor of family medicine at Temple University who is not involved in Bella's care, told CBS Philly.

McDonough said that even a common cold can be deadly for a child with the disorder. He said that since the Bella is over 3 years old, she's probably undergone a great deal of medical care up until now.

"Going to the hospital certainly is not something that would be unexpected," McDonough said, "but every time a child goes to the hospital with Trisomy-18, you worry a great deal."

CBS News reported that on Sunday Rick Santorum said Bella had a "miraculous turnaround" and remains in the hospital.

The Trisomy 18 Foundation has more on the genetic disorder.

Visit link:
Santorum's baby puts spotlight on genetic disorder

Recommendation and review posted by Bethany Smith

CHARLEROI, Belgium--(BUSINESS WIRE)-- The Belgian Biotech Company Delphi Genetics SA is proud to announce the launch of a newly-funded project. Together with academic and Biotech key-players, the company will participate in the development of DNA vaccines using the Staby® technology (antibiotic-free) during the next 3 years. The objective of the project funded by the Walloon region (BioWin project, 2.3 M €) is to develop and produce antibiotic-free DNA vaccines targeting some veterinary diseases.

The project also involves Eurogentec SA, another Belgian Biotech company (part of Kaneka) in charge of large scale DNA production and purification, and two universities: the Catholic University of Louvain in charge of the pharmaceutical and toxicity aspects of the project and the University of Liège in charge of the vaccinology and veterinary issues. “All partners complement one another perfectly” said Cédric Szpirer, CEO and Head of R&D of Delphi Genetics SA and explained:

“Today vaccination is an uncontested way of fighting disease. DNA vaccination seems to be a particularly promising method at this time, especially in the case of veterinary diseases. However, antibiotic-resistance genes are conventionally used during the construction of DNA vaccines but the resistance is increasingly less tolerated by regulatory agencies (FDA, USDA and EMA). In the context of this project, we propose to replace the antibiotic-resistance gene by the Staby® technology developed by Delphi Genetics and already used for production of recombinant proteins (higher yields and no antibiotics). In order to show the efficiency of our technology, we will develop new veterinary vaccines, we will validate that the method is usable for high scale DNA production and we will show its innocuousness.”

About Delphi Genetics

Founded at the end of 2001, Delphi Genetics SA develops technologies for genetic engineering and protein expression by using unique expertise in the domain of plasmid stabilisation systems.

Since 2004, Delphi Genetics has been marketing innovative kits for researchers. Some of these kits contain technologies that have since been licensed for industrial applications; in 2009 Delphi Genetics announced a non-exclusive licence agreement with Sanofi-Pasteur, the human vaccine division of Sanofi and a non-exclusive agreement with GSK in 2010. These agreements allow Sanofi-Pasteur and GSK to apply the StabyExpress® technology in the production of recombinant proteins, thus enabling them to produce a high yield without using antibiotics.

For more information, visit our website: http://www.delphigenetics.com

More:
Delphi Genetics: The New DNAVAC Research Project Targets the Removal of Antibiotics in Veterinary DNA-Vaccine ...

Recommendation and review posted by Bethany Smith

Of all the body’s organs, the human heart is probably the one most primed for
performance and efficiency. Decade after decade, it continues
to pump blood around our bodies. However, this performance
optimisation comes at a high price: over the course of
evolution, almost all of the body’s own regeneration mechanisms
in the heart have become deactivated. As a result, a heart
attack is a very serious event for patients; dead cardiac cells
are irretrievably lost. The consequence of this is a permanent
deterioration in the heart’s pumping power and in the patient’s
quality of life.

In their attempt to develop a treatment for the repair of
cardiac tissue, scientists are pursuing the
aim of growing replacement tissue in the laboratory, which
could then be used to produce replacement patches for the
repair of damaged cardiac muscle. The reconstruction of a
three-dimensional structure poses a challenge here. Experiments
have already been carried out with many different materials
that could provide a scaffold substance for the loading of cardiac muscle cells.

“Whether natural or artificial in origin, all of the tested
fibres had serious disadvantages,” says Felix Engel, Research
Group Leader at the Max Planck Institute for Heart and Lung
Research in Bad Nauheim. “They were either too brittle, were
attacked by the immune system or did not enable the heart
muscle cells to adhere correctly to the fibres.” However, the
scientists have now found a possible solution in Kharagpur,
India.

At the university there, coin-sized disks are being produced
from the cocoon of the tasar silkworm (Antheraea mylitta). According to
Chinmoy Patra, an Indian scientist who now works in Engel’s
laboratory, the fibre produced by the tasar silkworm displays
several advantages over the other substances tested. “The
surface has protein structures that facilitate the adhesion of
heart muscle cells. It’s also coarser than other silk fibres.”
This is the reason why the muscle cells grow well on it and can
form a three-dimensional tissue structure. “The communication
between the cells was intact and they beat synchronously over a
period of 20 days, just like real heart muscle,” says Engel.

Despite these promising results, clinical application of the
fibre is not currently on the agenda. “Unlike in our study,
which we carried out using rat cells, the problem of obtaining
sufficient human cardiac cells as starting material has not yet
been solved,” says Engel. It is thought that the patient’s own
stem cells could be used as starting material to avoid
triggering an immune reaction. However, exactly how the
conversion of the stem cells into cardiac muscle cells works
remains a mystery.

More information: Chinmoy Patra, Sarmistha Talukdar,
Tatyana Novoyatleva, Siva R. Velagala, Christian Mühlfeld,
Banani Kundu, Subhas C. Kundu, Felix B. Engel
Silk protein fibroin from Antheraea mylitta for cardiac tissue
engineering, Biomaterials, Advance Online Publication
Januar 10, 2012

Provided by Max-Planck-Gesellschaft (news : web)

Continue reading here:
Scientists use silk from the tasar silkworm as a scaffold for heart tissue

Recommendation and review posted by sam

Melbourne, Jan 30 (ANI): In a startling medical breakthrough,
scientists in Scotland have created brain tissue from skin samples of
patients who are suffering from mental illnesses such as
schizophrenia and depression.

The latest achievement was made by researchers at
Edinburgh's Centre for Regenerative
Medicine.

"A patient's neurones can tell us a great deal about the
psychological conditions that affect them, but you cannot
stick a needle in someone's brain and take out its cells,"
the Daily Telegraph quoted Professor Charles ffrench-Constant, the
center's director, as telling the Guardian.

"However, we have found a way round that. We can take a skin
sample, make stem cells from it and then direct
these stem cells to grow into brain cells. Essentially, we are
turning a person's skin cells into brain," he stated.

The scientists hope that studying these manufactured brain
cells will reveal clues to the conditions of patients with
mental illnesses - a task that had been challenging in the
past.

"It is very difficult to get primary tissue to study until
after a patient has died," said the Royal Edinburgh Hospital's
Professor Andrew McIntosh, who is collaborating with the
center on the project.

"Even then, that tissue is affected by whatever killed them
and by the impact of the medication they had been taking for
their condition, possibly for several decades. So having
access to living brain cells is a significant development for
the development of drugs for these conditions," McIntosh
added.

If successful, the same methods could be used for other
organs, including the liver and heart. (ANI)

See the rest here:
Human brain cells created from skin samples

Recommendation and review posted by sam

Sixteen years after Dolly the sheep was cloned in Edinburgh,
scientists in Scotland have made another startling medical
breakthrough.

Researchers at Edinburgh's Centre for Regenerative Medicine
have created brain tissue from patients suffering mental
illnesses such as schizophrenia and depression.

"A patient's neurones can tell us a great deal about the
psychological conditions that affect them, but you cannot stick
a needle in someone's brain and take out its cells," the
center's director, Professor Charles ffrench-Constant, told the
Guardian.

"However, we have found a way round that. We can take a skin
sample, make stem cells from it and then direct these stem
cells to grow into brain cells. Essentially, we are turning a
person's skin cells into brain."

The scientists hope that studying these manufactured brain
cells will reveal clues to the conditions of patients with
mental illnesses—a task that had been challenging in the past.

"It is very difficult to get primary tissue to study until
after a patient has died," said the Royal Edinburgh Hospital's
Professor Andrew McIntosh, who is collaborating with the center
on the project.

"Even then, that tissue is affected by whatever killed them and
by the impact of the medication they had been taking for their
condition, possibly for several decades. So having access to
living brain cells is a significant development for the
development of drugs for these conditions," McIntosh said.

If successful, the same methods could be used for other organs,
including the liver and heart.

Click here to read more. 

Read more from the original source:
Scientists use skin samples to create human brain cells

Recommendation and review posted by sam