PUBLIC RELEASE DATE: 29-Oct-2014 Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center @sumedicine The ability to pop a working copy of a faulty gene into a patient's genome is a tantalizing goal for many clinicians treating genetic diseases. Now, researchers at the Stanford University School of Medicine have devised a new way to carry out this genetic sleight of hand. The approach differs from that of other hailed techniques because it doesn't require the co-delivery of an enzyme called an endonuclease to clip the recipient's DNA at specific locations. It also doesn't rely on the co-insertion of genetic "on" switches called promoters to activate the new gene's expression. These differences may make the new approach both safer and longer-lasting. Using the technique, the Stanford researchers were able to cure mice with hemophilia by inserting a gene for a clotting factor missing in the animals. "It appears that we may be able to achieve lifelong expression of the inserted gene, which is particularly important when treating genetic diseases like hemophilia and severe combined immunodeficiency," said Mark Kay, MD, PhD, professor of pediatrics and of genetics. "We're able to do this without using promoters or nucleases, which significantly reduces the chances … Continue reading →

PUBLIC RELEASE DATE: 29-Oct-2014 Contact: Pete Farley peter.farley@ucsf.edu 415-502-6397 University of California - San Francisco @ucsf Scores of autoimmune diseases afflicting one in 12 Americans ranging from type 1 diabetes, to multiple sclerosis (MS), to rheumatoid arthritis, to asthma mysteriously cause the immune system to harm tissues within our own bodies. Now, a new study pinpoints the complex genetic origins for many of these diseases, a discovery that may lead to better diagnosis and ultimately to improved treatments. A team of scientists from UC San Francisco, the Broad Institute of MIT and Harvard, and Yale School of Medicine developed a new mathematical tool to more deeply probe existing DNA databases. In so doing they discovered how certain DNA variations, when inherited, are likely to contribute to disease. By applying their method to analyzing data from previous studies of 21 different autoimmune diseases, the research team has deepened scientific understanding of the genetic underpinnings of a wide range of these disorders. They also found the specific immune cells most responsible for the diseases. Their study is published online on October 29, 2014 in Nature. The researchers examined a wealth of data from 39 large-scale studies called genome-wide association studies (GWAS). Teams … Continue reading →

Contact Information Available for logged-in reporters only Newswise Scores of autoimmune diseases afflicting one in 12 Americans ranging from type 1 diabetes, to multiple sclerosis (MS), to rheumatoid arthritis, to asthma mysteriously cause the immune system to harm tissues within our own bodies. Now, a new study pinpoints the complex genetic origins for many of these diseases, a discovery that may lead to better diagnosis and ultimately to improved treatments. A team of scientists from UC San Francisco, the Broad Institute of MIT and Harvard, and Yale School of Medicine developed a new mathematical tool to more deeply probe existing DNA databases. In so doing they discovered how certain DNA variations, when inherited, are likely to contribute to disease. By applying their method to analyzing data from previous studies of 21 different autoimmune diseases, the research team has deepened scientific understanding of the genetic underpinnings of a wide range of these disorders. They also found the specific immune cells most responsible for the diseases. Their study is published online on October 29, 2014 in Nature. The researchers examined a wealth of data from 39 large-scale studies called genome-wide association studies (GWAS). Teams of scientists in recent years have conducted GWAS … Continue reading →

The U.S. Food and Drug Administration today announced it has awarded 15 grants totaling more than $19 million to boost the development of medical device, drug, and biological products for patients with rare diseases, with at least a quarter of the funding going to studies focused solely on pediatrics. The FDA awards grants for clinical studies on safety and/or effectiveness of products that could either result in, or substantially contribute to, approval of the products. The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program, said Gayatri R. Rao, M.D., director of the FDAs Office of Orphan Product Development. The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options. The program is administered through the FDAs Orphan Products Grants Program. This program was created by the Orphan Drug Act, passed in 1983, to promote the development of products for rare diseases. Since its inception, the program has given more than $330 million to fund more than 530 new clinical studies on developing treatments for rare … Continue reading →

Ras Al Khaimah, UAE (PRWEB) October 30, 2014 Grace Century portfolio project, Provia Laboratories, announced the appointment of Dr. James A. Manganello, Chairman of the Board, to the National Stem Cell Ethics Committee (NSCEC) of The Bahamas. The appointment was made by the Ministry of Health on behalf of the Government of The Bahamas on September 24th, 2014. More than a year after Bahamian Parliament passed the Stem Cell Therapy and Research Bill, Prime Minister Perry Christie said the government has completed the accompanying regulations and expects to begin approving applications for stem cell centers this month. Dr. Gomez, Minister of Health, said that the regulation of the stem cell therapy industry will lessen the potential for abuse and will ensure the highest standards of research and treatments are adhered to. Three committees have been established; the National Stem Cell Ethics Committee (NSCEC), the Scientific Committee, and the Compliance Committee. I am truly honored and delighted to be appointed to the NSCEC, and look forward to being part of the Bahamian Stem Cell Research and Therapy undertaking, said Dr. James Manganello. As Provia continues to expand its reach outside the US, I feel it is important to share our experience … Continue reading →