Archive for the ‘Gene Therapy Research’ Category
Gender influences symptoms of genetic disorder
PUBLIC RELEASE DATE:
6-Feb-2014
Contact: Michael C. Purdy 314-286-0122 Washington University School of Medicine
A genetic disorder that affects about 1 in every 2,500 births can cause a bewildering array of clinical problems, including brain tumors, impaired vision, learning disabilities, behavioral problems, heart defects and bone deformities. The symptoms and their severity vary among patients affected by this condition, known as neurofibromatosis type 1 (NF1).
Now, researchers at Washington University School of Medicine in St. Louis have identified a patient's gender as a clear and simple guidepost to help health-care providers anticipate some of the effects of NF1. The scientists report that girls with NF1 are at greater risk of vision loss from brain tumors. They also identified gender-linked differences in male mice that may help explain why boys with NF1 are more vulnerable to learning disabilities.
"This information will help us adjust our strategies for predicting the potential outcomes in patients with NF1 and recommending appropriate treatments," said David H. Gutmann, MD, PhD, the Donald O. Schnuck Family Professor of Neurology, who treats NF1 patients at St. Louis Children's Hospital.
The findings appear online in the Annals of Neurology.
Kelly Diggs-Andrews, PhD, a postdoctoral research associate in Gutmann's laboratory, reviewed NF1 patient data collected at the Washington University Neurofibromatosis (NF) Center. In her initial assessment, Diggs-Andrews found that the number of boys and girls was almost equal in a group of nearly 100 NF1 patients who had developed brain tumors known as optic gliomas. But vision loss occurred three times more often in girls with these tumors.
With help from David Wozniak, PhD, research professor of psychiatry, the scientists looked for an explanation in Nf1 mice (which, like NF1 patients, have a mutation in their Nf1 gene). They found that more nerve cells died in the eyes of female mice, and they linked the increased cell death to low levels of cyclic AMP, a chemical messenger that plays important roles in nerve function and health in the brain. In addition, Wozniak discovered that only female Nf1 mice had reduced vision, paralleling what was observed in children with NF1.
Two previous studies have shown that boys with NF1 are at higher risk of learning disorders than girls, including spatial learning and memory problems. To look for the causes of this gender-related difference, the scientists first confirmed that Nf1 mice had learning problems by testing the ability of the mice to find a hidden platform after training. After multiple trials, female Nf1 mice quickly found the hidden platform. In striking contrast, the male Nf1 mice did not, revealing that they had deficits in spatial learning and memory.
Researchers use genetic signals affecting lipid levels to probe heart disease risk
PUBLIC RELEASE DATE:
7-Feb-2014
Contact: John Ascenzi Ascenzi@email.chop.edu 267-426-6055 Children's Hospital of Philadelphia
New genetic evidence strengthens the case that one well-known type of cholesterol is a likely suspect in causing heart disease, but also casts further doubt on the causal role played by another type. The findings may guide the search for improved treatments for heart disease.
Most of us have heard of "good cholesterol" and "bad cholesterol" coursing through our bloodstream. In the conventional health wisdom of the past 30 years, having more of the "good" variety (high-density lipoprotein, or HDL) lowers your risk of heart disease, while more of the bad one (low-density lipoprotein, LDL) increases your risk. Indeed, over the years, clinical trials and other studies have found that drugs that lower LDL also lower your probability of heart disease.
On the other hand, drug trials have not shown heart-health benefits to increasing HDL or to lowering triglycerides, a third type of blood lipid. Now a new study co-led by scientists at The Children's Hospital of Philadelphia and Penn Medicine sheds light on the role of genes and blood lipid levels in cardiovascular health. Newer tools for gene analysis show how variations in DNA are underlying actors affecting heart diseasea major worldwide cause of death and disability.
"Now we are able to pinpoint gene signals that actually cause some of these conditions," says geneticist Brendan J. Keating, D. Phil., of The Center for Applied Genomics at The Children's Hospital of Philadelphia. "Unraveling how genetic variants that influence lipid traits are related to heart disease risk is a step toward designing treatments." Keating and his colleagues, working in large international collaborative groups, are wielding advanced gene-analysis tools to uncover important clues to heart disease.
Keating collaborated with clinical epidemiologist Michael V. Holmes, M.D., Ph.D., of the Perelman School of Medicine at the University of Pennsylvania, in a blood lipid study published online Jan. 27 in the European Heart Journal. Research co-authors were from six countries and various centers, including the University College London in the U.K.
The study team used a recently developed epidemiology tool called Mendelian randomization (MR). MR analyzes genetic variations using a method that identifies genes responsible for particular diseases, independent of confounding factors such as differences in behavior or environmental influences that often limit the conclusions of epidemiology research. This was one of the largest studies to date using MR, as well as the largest to use an allele-score method, described below.
The researchers analyzed DNA data from 17 studies including over 60,000 individuals, of whom more than 12,000 had experienced coronary heart disease, including heart attacks. Because previous studies had found signals from nearly 200 genes to be associated with blood lipid levels, the study team aggregated data into composite groups, called allele scores, for each of three blood lipids: LDL, HDL and triglycerides, then calculated their relationship to coronary heart disease.
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Researchers use genetic signals affecting lipid levels to probe heart disease risk
WorldCanvass: Genetics and New Technologies – February 15, 2013 – Video
WorldCanvass: Genetics and New Technologies - February 15, 2013
The study of genetics has come a long way since Gregor Mendel #39;s groundbreaking work with pea plants in the mid-19th century. To see just how far we #39;ve come a...
By: University of Iowa
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WorldCanvass: Genetics and New Technologies - February 15, 2013 - Video
The Sims 3 Perfect Genetics Challenge Oynuyoruz! – (CAS) Blm 1 – Mavi Sa Gzeldir! – Video
The Sims 3 Perfect Genetics Challenge Oynuyoruz! - (CAS) Blm 1 - Mavi Sa Gzeldir!
lk olarak erkek yattm karakterin dezavantajlarn fark edince, kz bir karakter yaratmaya karar verdim. Hem de "mavi" sal! 🙂 ^^ Siyah ekran olan kiil...
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The Sims 3 Perfect Genetics Challenge Oynuyoruz! - (CAS) Blm 1 - Mavi Sa Gzeldir! - Video
Stem Cell Therapy for Feline Kidney Disease, a Video Testimonial by a Pleased Pet Owner Gives Hope for Cats Suffering …
Poway, CA (PRWEB) February 06, 2014
Stem Cell Therapy for Feline Kidney Disease is a special interest piece produced by Nicky Sims, the owner of Kitters, who recently had Vet-Stem Regenerative Cell Therapy for his Feline Kidney Disease. Nicky highlights Kitters journey through diagnosis of the disease and his recent stem cell therapy, as well as educating about stem cells and their benefits.
Nickys film explains that Kitters began showing signs of kidney failure at the age of 15, exhibiting classic symptoms; lack of appetite, excessive thirst, nausea and lethargy. In 2012, Kitters was officially diagnosed with Chronic Renal Failure, or kidney disease. He was prescribed a low protein diet and subcutaneous fluids for rehydration. This has been the standard treatment for decades although it has only been shown to slow the progression of the disease; not reverse it.
Dr. Richter at Montclair Veterinary Hospital thinks that there is something else that can help. In recent years, his hospital has begun using stem cells to treat animals for various orthopedic conditions such as pain from arthritis and dysplasia. In October 2013, Kitters would be the first cat he had treated with stem cell therapy for Feline Kidney Disease.
Dr. Richter explains why this could work for Kitters, Stem cells are cells within your body that are able to turn into any other cell in the body. Kitters has kidney issues, so what weve done is harvested some fat from his abdomen and sent that fat to Vet-Stem in San Diego, and what they do is isolate the stem cells from the fatty tissue. They concentrate them and send them back to us. In the case of an animal with kidney disease, we just give the stem cells intravenously. What that is going to do is begin the healing and rebuilding process.
Nickys film explores the importance of kidneys stating they play a vital role, ridding the body of toxins. As kidney disease progresses scar tissue develops making it harder to filter toxins. Damage to the kidneys makes the animal vulnerable to a number of other health conditions. Unfortunately the disease usually goes undiagnosed given that the symptoms of the disease often do not show until 2/3 of the kidneys are damaged.
Kitters own stem cells were used with the hope of repairing his damaged tissue Dr. Richter goes on, The nice thing about stem cells is that there is no issue of tissue rejection, since it is Kitters own stem cells. Additionally, if there is anything else going on in his body beyond the kidneys its going to address that as well. So, it is a really wonderful systemic treatment.
To find out more or view the special interest piece by Nicky Sims, Stem Cell Therapy for Feline Kidney Disease, visit this link.
About Vet-Stem, Inc. Vet-Stem, Inc. was formed in 2002 to bring regenerative medicine to the veterinary profession. The privately held company is working to develop therapies in veterinary medicine that apply regenerative technologies while utilizing the natural healing properties inherent in all animals. As the first company in the United States to provide an adipose-derived stem cell service to veterinarians for their patients, Vet-Stem, Inc. pioneered the use of regenerative stem cells in veterinary medicine. The company holds exclusive licenses to over 50 patents including world-wide veterinary rights for use of adipose derived stem cells. In the last decade over 10,000 animals have been treated using Vet-Stem, Inc.s services, and Vet-Stem is actively investigating stem cell therapy for immune-mediated and inflammatory disease, as well as organ disease and failure. For more on Vet-Stem, Inc. and Veterinary Regenerative Medicine visit http://www.vet-stem.com or call 858-748-2004.
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Stem Cell Therapy for Feline Kidney Disease, a Video Testimonial by a Pleased Pet Owner Gives Hope for Cats Suffering ...
Research and Markets: RNAi – Technologies, Markets and Companies – 2014 Report
DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/dlx7gv/rnai) has announced the addition of Jain PharmaBiotech's new report "RNAi - Technologies, Markets and Companies" to their offering.
RNA interference (RNAi) or gene silencing involves the use of double stranded RNA (dsRNA). Once inside the cell, this material is processed into short 21-23 nucleotide RNAs termed siRNAs that are used in a sequence-specific manner to recognize and destroy complementary RNA. The report compares RNAi with other antisense approaches using oligonucleotides, aptamers, ribozymes, peptide nucleic acid and locked nucleic acid.
Various RNAi technologies are described, along with design and methods of manufacture of siRNA reagents. These include chemical synthesis by in vitro transcription and use of plasmid or viral vectors. Other approaches to RNAi include DNA-directed RNAi (ddRNAi) that is used to produce dsRNA inside the cell, which is cleaved into siRNA by the action of Dicer, a specific type of RNAse III. MicroRNAs are derived by processing of short hairpins that can inhibit the mRNAs. Expressed interfering RNA (eiRNA) is used to express dsRNA intracellularly from DNA plasmids.
Delivery of therapeutics to the target tissues is an important consideration. siRNAs can be delivered to cells in culture by electroporation or by transfection using plasmid or viral vectors. In vivo delivery of siRNAs can be carried out by injection into tissues or blood vessels or use of synthetic and viral vectors.
Because of its ability to silence any gene once the sequence is known, RNAi has been adopted as the research tool to discriminate gene function. After the genome of an organism is sequenced, RNAi can be designed to target every gene in the genome and target for specific phenotypes. Several methods of gene expression analysis are available and there is still need for sensitive methods of detection of gene expression as a baseline and measurement after gene silencing. RNAi microarray has been devised and can be tailored to meet the needs for high throughput screens for identifying appropriate RNAi probes. RNAi is an important method for analyzing gene function and identifying new drug targets that uses double-stranded RNA to knock down or silence specific genes. With the advent of vector-mediated siRNA delivery methods it is now possible to make transgenic animals that can silence gene expression stably. These technologies point to the usefulness of RNAi for drug discovery.
RNAi can be rationally designed to block the expression of any target gene, including genes for which traditional small molecule inhibitors cannot be found. Areas of therapeutic applications include virus infections, cancer, genetic disorders and neurological diseases. Research at academic centers that is relevant to RNAi-based therapeutics is mentioned.
Regulatory, safety and patent issues are discussed. Side effects can result from unintended interaction between an siRNA compound and an unrelated host gene. If RNAi compounds are designed poorly, there is an increased chance for non-specific interaction with host genes that may cause adverse effects in the host. However, there are no major safety concerns and regulations are in preliminary stages as the clinical trials are still ongoing and there are no marketed products. Many of the patents are still pending.
The markets for RNAi are difficult to define as no RNAi-based product is approved yet but several are in clinical trials. The major use of RNAi reagents is in research but it partially overlaps that of drug discovery and therapeutic development. Various markets relevant to RNAi are analyzed from 2013 to 2023. Markets are also analyzed according to technologies and use of siRNAs, miRNAs, etc.
Profiles of 161 companies involved in developing RNAi technologies are presented along with 233 collaborations. They are a mix of companies that supply reagents and technologies (nearly half of all) and companies that use the technologies for drug discovery. Out of these, 33 are developing RNAi-based therapeutics and 35 are involved in microRNAs. The bibliography contains selected 600 publications that are cited in the report. The text is supplemented with 37 tables and 11 figures.
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Research and Markets: RNAi - Technologies, Markets and Companies - 2014 Report
New disease gene discovery sheds light on cause of bone marrow failure
PUBLIC RELEASE DATE:
6-Feb-2014
Contact: Charli Scouller c.scouller@qmul.ac.uk 07-709-825-741 Queen Mary, University of London
The study, published in The American Journal of Human Genetics, detected and identified a new disease gene (ERCC6L2). In its normal form, the gene plays a key role in protecting DNA from damaging agents, but when the gene is mutated the cell is not able to protect itself in the normal way.
The research findings suggest that the gene defect and the subsequent DNA damage was the underlying cause of bone marrow failure among the study participants.
Bone marrow failure is a term used for a group of life threatening disorders associated with an inability of the bone marrow to make an adequate number of mature blood cells.
Patients were recruited from all over the world to join an international bone marrow failure registry and researchers used new DNA sequencing technologies to study cases of bone marrow failure with similar clinical features. These included bone marrow failure associated with neurological abnormalities (learning defects and developmental delay), and patients whose parents were first cousins.
The findings mean it is now possible to carry out a reliable genetic test (including antenatal testing) in these families and get an accurate diagnosis. In the long term, with further research, the findings could lead to the development of new treatment for this specific gene defect.
Professor Inderjeet Dokal, Chair of Paediatrics and Child Health at Queen Mary University of London, comments:
"New DNA sequencing technology has enabled us to identify and define a new gene defect which causes a particular type of bone marrow failure. This is a promising finding which we hope one day could lead to finding an effective treatment for this type of gene defect. Clinicians treating patients with bone marrow failure should now include analysis for this gene in their investigation.
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New disease gene discovery sheds light on cause of bone marrow failure
First FSH Muscular Dystrophy High-Throughput Drug Discovery Study Published
Lexington, MASS (PRWEB) February 06, 2014
Facioscapulohumeral muscular dystrophy (FSHD) is among the most common muscle-wasting diseases, affecting more than 500,000 people around the world. Its cause is genetic, passed from generation to generation, although 30 percent of cases arise spontaneously in families with no previous history. There is no treatment, but in a new study published in the journal, Skeletal Muscle on February 1, 2014, researchers have identified dozens of compounds showing early promise for future treatments.
This is the first published high-throughput drug screening study for FSHD, noted June Kinoshita, Executive Director of the FSH Society, which helped to fund the research. Years of investment in basic research to understand the genetic mechanism of the disease and to develop cell-based assays have made it possible to carry out this efficient strategy to identify drug candidates.
Recent discoveries point to a mysterious protein called DUX4 as a lead suspect in causing FSHD. Normally suppressed in adult muscles, DUX4 is unleashed in FSHD, with toxic effects on muscle cells. In people with FSHD, the facial (facio), shoulder (scapula) and upper arm (humeral) muscles are prone to degenerate, giving the disease its name. FSHD can also affect the lower abdomen and legs, leading to profound disability.
To hunt for drugs that can stop DUX4, the research team, led by Michael Kyba, PhD, of the University of Minnesota, engineered mouse myoblasts (immature muscle cells) to express DUX4 under the control of a genetic switch that is triggered by adding the antibiotic doxycycline to the petri dish.
When DUX4 is switched on, the cells begin to die, and they also become more vulnerable to a variety of chemical insults. Drugs were added to the cultured cells to see if any of them rescued the cells.
The research team tested thousands of compounds on these DUX4-expressing cells, including 1,120 Food and Drug Administration-approved off-patent drugs and 43,000 other chemicals. After extensive studies to weed out false positives, and further winnowing to select compounds with favorable chemical properties, the investigators honed the list to 52 hits, or candidates.
Remarkably, two-thirds of our hits are compounds that protect cells from oxidative stress, Kyba said. Although we need to be cautious extrapolating from cells in a dish to human patients, I am enthusiastic about testing whether protecting cells from oxidative stress is beneficial in FSHD.
Further work is now being planned to understand the precise mechanism behind the anti-DUX4 activity of each of these compounds. This research will help the investigators focus on the most promising ones to develop into therapies. At the same time, each compound can bring to light new insights into how DUX4 causes this devastating disease.
We were very, very fortunate to get support from the FSH Society in the form of a postdoctoral fellowship back in the dark ages when nobody else was funding research on DUX4, said Kyba. Additional funding came from the National Institutes of Health, Dr. Bob and Jean Smith Foundation, Friends of FSH Research, the FSHD Global Research Foundation and the Muscular Dystrophy Association.
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First FSH Muscular Dystrophy High-Throughput Drug Discovery Study Published
Critical factor (BRG1) identified for maintaining stem cell pluripotency
PUBLIC RELEASE DATE:
6-Feb-2014
Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News
New Rochelle, NY, February 6, 2014The ability to reprogram adult cells so they return to an undifferentiated, pluripotent statemuch like an embryonic stem cellis enabling the development of promising new cell therapies. Accelerating progress in this field will depend on identifying factors that promote pluripotency, such as the Brg1 protein described in a new study published in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResearch Open Access website.
In "BRG1 Is Required to Maintain Pluripotency of Murine Embryonic Stem Cells," Nishant Singhal and coauthors, Max Planck Institute for Molecular Biomedicine, Mnster, and University of Mnster, Germany, demonstrate the critical role the Brg1 protein plays in regulating genes that are part of a network involved in maintaining the pluripotency of embryonic stem cells. This same network is the target for methods developed to reprogram adult somatic cells.
"This work further clarifies the role of the Brg1 containing BAF complex in regulating pluripotency and has important implications for all cellular reprogramming technologies," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland.
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About the Journal
About the Publisher
Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in promising areas of science and biomedical research, including, DNA and Cell Biology, Tissue Engineering, Stem Cells and Development, Human Gene Therapy, HGT Methods, and HGT Clinical Development, and AIDS Research and Human Retroviruses. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.
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Critical factor (BRG1) identified for maintaining stem cell pluripotency
Public divided on genetic testing to predict cancer risk …
A national poll from the University of Utah's Huntsman Cancer Institute shows 34 percent of respondents would not seek genetic testing to predict their likelihood of developing a hereditary cancer -- even if the cost of the testing was not an issue. Concerns about employment and insurability were cited as the primary reason, even though current laws prohibit such discrimination.
The poll also shows only 35 percent of respondents would be extremely or very likely to seek aggressive prophylactic or preventive treatment, such as a mastectomy, if they had a family history of cancer and genetic testing indicated a genetic pre-disposition to cancer.
"I see patients every week who could have taken steps to reduce their risk if they'd known they'd had a predisposition for a certain type of cancer. The best treatment for cancer is prevention, of which genetic testing plays an integral role," said Saundra Buys, M.D., co-director of the Family Cancer Assessment Clinic and medical director of the High Risk Cancer Research at Huntsman Cancer Institute (HCI), and professor of medicine at the University of Utah. "In addition to educating the public about the important role genetic testing plays in both prevention and treatment of cancer, we must also work to eliminate perceived false barriers to testing, such as concerns about insurability and employment."
Nearly 40 percent of those who said they wouldn't seek testing reported being somewhat or extremely concerned that the results would impact opportunities for employment, while 69 percent of that same group reported being somewhat or extremely concerned that the results would have an adverse impact on their ability to get insurance.
Inherited mutations play a major role in the development of approximately 5 percent of all cancers. Genetic mutations associated with more than 50 hereditary cancer syndromes -- including those discovered at the University of Utah for melanoma, colon and breast cancer -- have been identified.
Buys says the survey demonstrates that even with increased media attention to genetic testing in recent months more work is needed to educate the public about the type of information genetic testing provides and who should seek it. She says family and personal health history are the most important factors in determining whether a person should consider genetic testing.
She warns, however, that genetic testing is only as good as the genetic counseling that accompanies it. "There are many genetic tests being ordered in physician offices around the country without the benefit of genetic counseling. The results of these tests are complex, and without appropriate counseling, can cause confusion and unneeded anxiety for patients," said Buys.
Other findings from the poll:
Following recommended screenings: 63 percent of respondents reported being extremely or very likely to follow all recommended screenings if they knew there was a history of cancer in their family.
Testing to help identify best course of treatment: 85 percent of respondents stated that if diagnosed with cancer they would be willing to undergo genetic testing if it could help determine the most effective course of treatment.
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Public divided on genetic testing to predict cancer risk ...
Genetics Hens , and the problem of eating there Eggs – Video
Genetics Hens , and the problem of eating there Eggs
By: Ray wasp
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Genetics Hens , and the problem of eating there Eggs - Video
Building Better Genetics – Schiefelbein Farms – Video
Building Better Genetics - Schiefelbein Farms
Cattlemen like Minnesota Angus breeder Don Schiefelbein rely on DNA information from the Zoetis HD 50K test and genomic-enhanced EPDs to make the best possib...
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Building Better Genetics - Schiefelbein Farms - Video
Introduction MODY and GDM Genetics – Video
Introduction MODY and GDM Genetics
Watch this movie on Plotagon: http://plotagon.com/3810.
By: Linda Adkison
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Introduction MODY and GDM Genetics - Video
Alliance for Cancer Gene Therapy (ACGT) Targets Brain, Pediatric Cancers with $1 Million in New Grants
Stamford, CT (PRWEB) February 06, 2014
Alliance for Cancer Gene Therapy (ACGT) the nations only non-profit dedicated exclusively to cell and gene therapies for cancer is redoubling its efforts to treat and combat cancers in the New Year, and announces $1 million in recent grants.
The funding spread across three grants will support basic and clinical science at premier institutions in and outside the United States, with ACGTs mission top-of-mind: uncovering effective, innovative cancer treatments that supersede radiation, chemotherapy and surgery.
This January, ACGTs President and Co-Founder Barbara Netter has announced two Young Investigator Grants that provide promising researchers with $250,000 each for two- to three-year studies.
Young Investigator Fan Yang, PhD Assistant Professor of Orthopedic Surgery and Bioengineering at Stanford University will use the funds to research new treatment options for pediatric brain cancer, the leading cause of death from childhood cancer. Dr. Yangs study will deploy adult-derived stem cells to target solid brain tumor cells, which are often highly invasive and difficult to treat.
Arnob Banerjee, MD, PhD Assistant Professor of Hematology and Oncology at the University of Maryland will use ACGTs funding to further develop the long-term effectiveness of immune-mediated treatments, the most advanced form of gene therapy.
It is imperative that the best and brightest young scientists like Fan Yang and Arnob Banerjee have the funds necessary to study and treat cancer, Netter said. This was my husband Edwards vision in 2001, when gene cell therapy was a fledgling science. We are proud to continue his pioneering foresight today. Partnerships with Dr. Yang, a former fellow at MIT, and Dr. Banerjee, a former fellow and instructor at the University of Pennsylvania, dovetail with ACGTs record of funding outstanding researchers and physicians with the capability to make unprecedented breakthroughs.
The Young Investigator grants come on the heels of a $500,000 Investigators Award to John Bell, PhD, Senior Research Scientist and Professor of Medicine at the Ottawa Hospital Research Institute in Canada. Dr. Bell has worked extensively with oncolytic viruses man-made viruses that target only cancer cells, and spare patients the harrowing side-effects of chemotherapy, radiation or surgery and has discovered the enormous promise they offer in the war on cancer.
The research and trials funded by ACGTs grant have the potential to treat metastatic and recurrent brain cancer, extend patients survival timeline, and vastly improve patients quality of life during treatment, Dr. Bell said.
ACGT has served as a major funding engine in the fight against cancer since its formation in 2001, and has provided nearly $25 million in grants to date. ACGT was created by Barbara and Edward Netter after the loss of their daughter-in-law to breast cancer. Since Edwards passing in 2011, Barbara Netter has led the foundation as President and Co-Founder, continuing her husbands vision.
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Alliance for Cancer Gene Therapy (ACGT) Targets Brain, Pediatric Cancers with $1 Million in New Grants
14 Month Results After Stem Cell Therapy by Dr Harry Adelson for Arthritic Hip – Video
14 Month Results After Stem Cell Therapy by Dr Harry Adelson for Arthritic Hip
http://www.docereclinics 14 months after stem cell therapy for his arthritic hip, Marty discusses his results by Dr. Harry Adelson. Call the clinic today at ...
By: Harry Adelson, N.D.
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14 Month Results After Stem Cell Therapy by Dr Harry Adelson for Arthritic Hip - Video
therapy treatment for stem cell therapy treatment for cerebral palsy by dr alok sharma, mumbai, – Video
therapy treatment for stem cell therapy treatment for cerebral palsy by dr alok sharma, mumbai,
improvement seen in just 5 days after stem cell therapy treatment for cerebral palsy by dr alok sharma, mumbai, india. Stem Cell Therapy done date 31 Dec 201...
By: Neurogen Brain and Spine Institute
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therapy treatment for stem cell therapy treatment for cerebral palsy by dr alok sharma, mumbai, - Video
Webinar: Reimbursement Strategies for Stem Cell Therapy Products – Video
Webinar: Reimbursement Strategies for Stem Cell Therapy Products
Planning Your Payment Strategy in Early Product Development EXPERT SPEAKERS: Michael Werner, J.D., Executive Director, Alliance for Regenerative Medicine;...
By: Fernanda Torres
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Webinar: Reimbursement Strategies for Stem Cell Therapy Products - Video
New stem cell research removes reliance on human and animal cells
13 hours ago
A new study, published today in the journal Applied Materials & Interfaces, has found a new method for growing human embryonic stem cells, that doesn't rely on supporting human or animal cells.
Traditionally, these stem cells are cultivated with the help of proteins from animals, which rules out use in the treatment of humans. Growing stem cells on other human cells risks contamination with pathogens that could transmit diseases to patients.
The team of scientists led by the University of Surrey and in collaboration with Professor Peter Donovan at the University of California have developed a scaffold of carbon nanotubes upon which human stem cells can be grown into a variety of tissues. These new building blocks mimic the surface of the body's natural support cells and act as scaffolding for stem cells to grow on. Cells that have previously relied on external living cells can now be grown safely in the laboratory, paving the way for revolutionary steps in replacing tissue after injury or disease.
Dr Alan Dalton, senior lecturer from the Department of Physics at the University of Surrey said: "While carbon nanotubes have been used in the field of biomedicine for some time, their use in human stem cell research has not previously been explored successfully."
"Synthetic stem cell scaffolding has the potential to change the lives of thousands of people, suffering from diseases such as Parkinson's, diabetes and heart disease, as well as vision and hearing loss. It could lead to cheaper transplant treatments and could potentially one day allow us to produce whole human organs without the need for donors."
Explore further: New method increases supply of embryonic stem cells
A new method allows for large-scale generation of human embryonic stem cells of high clinical quality. It also allows for production of such cells without destroying any human embryos. The discovery is a big step forward ...
A research team has identified a protein that increases the transfer of mitochondria from mesenchymal stem cells to lung cells. In work published in The EMBO Journal, the researchers reveal that the delivery of mitochondria to hum ...
Abba Zubair, M.D., Ph.D, believes that cells grown in the International Space Station (ISS) could help patients recover from a stroke, and that it may even be possible to generate human tissues and organs in space. He just ...
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New stem cell research removes reliance on human and animal cells
Inner workings of a cellular nanomotor revealed
20 hours ago In this rendering of the SecA nanomotor, the two pistons (colored cyan and light green) await the arrival of a protein. Cylinders, arrows and loops make up the nanomotor's mechanical parts. Credit: T. Economou
Our cells produce thousands of proteins but more than one-third of these proteins can fulfill their function only after migrating to the outside of the cell. While it is known that protein migration occurs with the help of various 'nanomotors' that push proteins out of the cell, little is known about their precise mechanical functioning. New research by Anastassios Economou (KU Leuven) and his team reveals the inner workings of one such nanomotor, called SecA, with new clarity.
Protein migration is a fundamental problem in biology and is essential for life. Examples of migrating proteins include insulin (the absence of which leads to diabetes), antibodies (essential for combating infections), membrane channels (essential for neuronal cell function) and toxin-proteins (secreted by pathogenic microorganisms).
Migrating proteins contain chemical signals called signal peptides. These signal peptides act as postal addresses and direct exported proteins to the membrane for transport outside the cell.
In previous research, Dr Economou, in collaboration with Babis Kalodimos (Rutgers University), revealed how signal peptides bind to a specific cellular receptor on the membrane, which subsequently connects to the export channel leading out of the cell. This receptor was also found to act as a nanomotor, with two separate piston-like mechanical parts that somehow push proteins out of the cell. The exact mechanism by which this occurred remained a mystery. Until now.
In the current study, published in the December issue of Molecular Cell, Economou and his team show how the SecA receptor moves to push proteins out of the cell: when a signal peptide makes contact, the two pistons become excited. They position themselves one against the other in a series of defined steps and modify their shape. This finely orchestrated series of motions opens the export channel and traps the exported protein inside it. In a final step, the two parts dissociate and the remaining single piston pushes the protein out in cycles of repeated motions.
The discovery adds a significant piece in the puzzle of exploiting protein migration to improve health. "Moving forward, this discovery will help us focus efforts to find specific antibiotics against harmful bacterial protein secretion pathways," says Dr Economou. "It also offers the possibility to optimise biotechnological production of human biopharmaceuticals by using microbial 'cell factories' for secreting biopharmaceuticals. We will be pursuing these avenues in future research."
Explore further: Designer proteins provide new information about the body's signal processes
More information: The paper "Quaternary Dynamics of the SecA Motor Drive Translocase Catalysis" by Giorgos Gouridis, Spyridoula Karamanou, Marios Frantzeskos Sardis, Martin Alexander Schrer, Guido Capitani and Anastassios Economou can be accessed on the website of Molecular Cell: http://www.cell.com/molecular-cell/abstract/S1097-2765(13)00860-5
Journal reference: Molecular Cell
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Inner workings of a cellular nanomotor revealed
Policymakers and scientists agree on top research questions
20 hours ago
Natural resource managers, policymakers and their advisers, and scientists have similar ideas about which research questions could, if answered, most increase the effectiveness of US natural resource management policies. A survey of over 600 members of those professional groups revealed how they ranked the importance of 40 management-relevant research questions identified in earlier work. Respondents judged the most important of the 40 questions to be the quantity and quality of surface and groundwater that will be necessary to sustain US human populations and ecosystem resilience during the next 100 years.
The Internet-based survey that yielded the rankings is described in an article to be published in the March issue of BioScience by Murray Rudd, of the University of York, United Kingdom, and Erica Fleishman, of the University of California, Davis.
The researchers used a technique called best-worst scaling to eliminate some of the biases that often confound rating studies: respondents repeatedly ranked small subsets of the 40 questions.
The questions had been identified earlier by natural resource managers, policymakers, and their advisers.
Statistical analysis of the survey results revealed the existence of subgroups with similar ideas about the importance of some questions, but there were no significant differences between policymakers and scientists, a result that surprised Rudd and Fleishman.
They are continuing to analyze results from this and a follow-up survey on related questions.
Explore further: The science of baby-making still a mystery for many women
More information: The earlier study identifying the 40 management-relevant research questions was published in BioScience in 2011 and can be read at bioscience.oxfordjournals.org/content/61/4/290.full
A wide-ranging group of experts has published a set of 40 key environmental questions to help align scientific research agendas with the needs of natural resource decision makers.
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Policymakers and scientists agree on top research questions
Research and Markets: Concise Analysis of the …
DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/7pg67k/gene_therapy) has announced the addition of a new report "Concise Analysis of the International Gene Therapy Market: Updated 2014 Report" to their offering.
Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.
Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.
Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2013, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.
Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency.
Key Topics Covered:
Technologies & Markets
Executive Summary
1. Introduction
2. Gene Therapy Technologies
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Gene that influences receptive joint attention in chimpanzees gives insight into autism
PUBLIC RELEASE DATE:
4-Feb-2014
Contact: Lisa Newbern lisa.newbern@emory.edu 404-727-7709 Emory Health Sciences
Following another's gaze or looking in the direction someone is pointing, two examples of receptive joint attention, is significantly heritable according to new study results from researchers at the Yerkes National Primate Research Center, Emory University. Determining such communicative cues are significantly heritable means variation in this ability has a genetic basis, which led the researchers to the vasopressin receptor gene, known for its role in social bonding.
The study results, which are published in Scientific Reports, give researchers insight into the biology of disorders in which receptive joint attention is compromised, such as autism spectrum disorders (ASD), and may ultimately lead to new diagnosis and treatment strategies.
According to Yerkes researchers Larry Young, PhD, and Bill Hopkins, PhD, co-authors of the study, receptive joint attention is important for developing complex cognitive processes, including language and theory of mind, and poor joint attention abilities may be a core feature in children with or at risk of developing ASD.
Young is division chief of Behavioral Neuroscience and Psychiatric Disorders at Yerkes, director of the Center for Translational Social Neuroscience (CTSN) at Emory and William P. Timmie Professor in the Emory University School of Medicine Department of Psychiatry and Behavioral Sciences. Yerkes researcher Hopkins is also a core faculty member in the Neuroscience Institute of Georgia State University and newly named science director of the Iowa Primate Learning Sanctuary.
Young and Hopkins led a collaborative team of researchers from Yerkes, the CTSN, the Neuroscience Institute at Georgia State University and the University of Texas M.D. Anderson Cancer Center. They studied chimpanzees to determine the extent to which the animals follow gaze or pointing by a human.
"We used chimpanzees in this behavioral study because their receptive joint attention abilities are well documented and their closeness to humans makes the study results the most likely to be generalizable to humans," says Hopkins.
Young's previous research in which he showed the vasopressin receptor gene was necessary for remembering individuals (or social memories) and for social bonding in male rodents was key to designing the current study. According to Young, variation in the length of a stretch of repetitive DNA, known as junk DNA, in the control region of the vasopressin receptor gene predicted if a male prairie vole was likely to form monogamous bonds with a mate. Human-based studies suggest that a similar repetitive element, referred to as RS3, in the control region of the human vasopressin receptor gene predicts romantic relationship quality and generosity.
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Gene that influences receptive joint attention in chimpanzees gives insight into autism
Grapevine gene bank under threat
Christophe Simon/AFP/Getty
The Domaine de Vassal vine collection near Montpellier holds 2,300 different grape varieties.
Uncertainty hangs over one of the worlds largest and most important grapevine collections. The Domaine de Vassal vineyard, on Frances Mediterranean coast, houses a vast sweep of grape biodiversity that is essential to research and winegrowers in France and around the world.
The 138-year-old collection, managed by the French National Institute for Agricultural Research (INRA), has been threatened with eviction, prompting a decision to relocate it.
That is raising concerns among scientists and winegrowers, because money to pay for the prospective move costing an estimated 4 million (US$5.4 million) has yet to be found. Even then, the sheer logistical complexity is such that relocation is likely to take years to complete, says INRA, and means that much of its research may be put on hold.
Dubbed the Louvre of grapevines by the local press, the vineyard near Marseillan, southwest of Montpellier, contains thousands of unique grape varieties. As well as having a conservation role in preserving genetic diversity, the collection is used for research and for breeding qualities such as flavour, colour, adaptation to specific regions and pathogen resistance. Several hundred samples from the Domaine de Vassal are used annually, mainly by other French labs, but also internationally.
The collection is of utmost value to the international grapevine genetics community, says Carole Meredith, an emeritus geneticist at the University of California, Davis. Although many countries have established collections of their own heritage grape varieties, the Vassal collection is among the oldest and best curated.
Meredith notes that much of her own research would have been impossible without this living library. Her labs previous studies of the vineyards specimens revealed Chardonnays somewhat undistinguished heritage one of its parent varieties is a noble Pinot, but the other is a Gouais, a grape long shunned as mediocre (J. Bowers etal. Science 285, 15621565; 1999).
The collection was started in 1876 by French researchers in response to a pest outbreak that saw the near-destruction of Europes vineyards. The outbreak was caused by accidental introduction of phylloxera an aphid that infests roots and kills the vine.
The vineyard was initially located near Montpellier, but moved to the Domaine de Vassal in 1949, where it expanded greatly. It now houses some 7,500 accessions from 47countries, representing 2,300 different grape varieties, including wild species, rootstocks, hybrids and mutants.
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Grapevine gene bank under threat