Category Archives: Gene Therapy

A Stem Cell Cure for Bubble Baby Disease (SCID) Visit: http://www.uctv.tv/) On November 18th, 2014, a UCLA research team led by Donald Kohn, M.D., announced a breakthrough gene therapy and stem cell cure ... By: University of California Television (UCTV) … Continue reading →

Cancer Gene Therapy -- 8News Anchor Amy Lacey/VCU Massey Cancer Center It can be devastating to be diagnosed with cancer, but what if you could take drugs tailored to your DNA, drugs that can buy you precious time? 8News Anchor ... By: Amy Lacey … Continue reading →

Gene therapy leads to promising avenue for HIV vaccine Gene therapy leads to promising avenue for HIV vaccine. By: 7BILLIONHUMANBEING … Continue reading →

Uptown Funk Gene Therapy Parody This is a video made for a 9th grade biology "celebration" grade Have fun??? By: Andrew Melton … Continue reading →

The Clinical Promise of the Gene Therapy from Harrison's Principles of IM, 19th Edition Dr. J. Larry Jameson, editor for Harrison's Principles of Internal Medicine, discusses the clinical promise of the gene therapy. Visit http://www.HarrisonsIM.com to watch more videos of Dr. J. Larry... By: McGrawHIllPro … Continue reading →

Uptown Funk Gene Therapy This video is about Uptown Funk Gene Therapy. By: Andrew Melton … Continue reading →

UD alumni $2.5 million closer to saving 5-year-old Eliza In 2014, University of Delaware alumni Glenn and Cara O'Neill raised half of the $5 million needed for a gene therapy clinical trial and the medicine necessa... By: UnivDelaware … Continue reading →

Feb 16, 2015 by Amanda Siegfried Dr. Leonidas Bleris (left), assistant professor of bioengineering at UT Dallas,and Richard Taplin Moore MS11 helped create a new delivery system that may change gene therapy. Bioengineers at The University of Texas at Dallas have created a novel gene-delivery system that shuttles a gene into a cell, but only for a temporary stay, providing a potential new gene-therapy strategy for treating disease. The approach offers distinct advantages over other types of gene therapies under investigation, said Richard Taplin Moore MS'11, a doctoral student in bioengineering in the Erik Jonsson School of Engineering and Computer Science. He is lead author of a study describing the new technique in the Jan. 30 issue of the journal Nucleic Acids Research. "In other gene therapy approaches, the therapeutic genetic messages being delivered can persist for a long time in the patient, potentially lasting for the patient's entire lifetime," Moore said. "This irreversibility is one reason gene therapies are so difficult to get approved." The UT Dallas study describes proof-of-concept experiments in which a gene carrying instructions for making a particular protein is ordered to self-destruct once the cell has "read" the instructions and made a certain quantity of … Continue reading →

Flow chart of one method of engineering T cells to fight cancer. Tough blood cancers are responding to treatment with the patient's own genetically engineered immune cells, according to a cancer specialist who is helping test the bold -- and risky -- approach. The treatment has produced complete remissions in large percentages of patients treated, up to 90 percent in one group of 30 patients. Moreover, these are all extremely sick patients, whose cancer has resisted other therapies, leaving them with virtually no options. The longest survivor has been in complete remission for more than 4 years, said Dr. David Porter of the University of Pennsylvania. Porter spoke Sunday at the 35th Annual Conference on Clinical Hematology & Oncology, held in La Jolla by Scripps Health. Speakers like Porter came from around the country to discuss advances in their field, part of a continuing medical education program to keep doctors up to date with the latest medical advances. The conference continues through Tuesday. Dr. David Porter / University of Pennsylvania Porter works with Dr. Carl June and other colleagues to hone the effectiveness of using T cells genetically programmed to attack malignant B cells. B cell malignancies cause such cancers … Continue reading →

Collaboration entails development of gene therapy treatment for severe neurodegenerative disease GM1-gangliosidosis LYSOGENE, a leading, clinical stage gene therapy biotechnology company committed to the development and commercialization of breakthrough treatments for severe orphan pathologies affecting the central nervous system (CNS), recently announced that it has entered into a strategic collaboration with the University of Massachusetts Medical School (UMMS) in Worcester, Massachusetts, and Auburn University (AU) in Auburn, Alabama. Through the collaboration, LYSOGENE, UMMS and AU will develop IND-supporting preclinical studies in GM1-gangliosidosis, a rare, inherited disorder characterized by severe neurological impairment, using adeno-associated virus (AAV) gene therapy technology. The collaboration will combine LYSOGENEs outstanding translational and clinical expertise in gene therapy for CNS disorders with the unique preclinical expertise and infrastructure of UMMS and AU to design and test innovative AAV-based gene therapy approaches to treat GM1-gangliosidosis. The development of a potential treatment for GM1-gangliosidosis using AAV gene therapy was initiated in 2005 by Miguel Sena-Esteves, PhD, associate professor in the Neurology Department and the Gene Therapy Center at UMMS, and Douglas R. Martin, PhD, associate professor in the Scott-Ritchey Research Center and Department of Anatomy, Physiology & Pharmacology at AU. The approach developed by the investigators uses AAV vectors … Continue reading →