Latest Adeno-associated virus Stories
Results bode well for treating people with the diseaseGene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from...
Tag-team Approach Breaks the Size Barrier for Gene TherapyNeuroscientists have forged an unlikely molecular union as part of their fight against diseases of the brain and nervous system.The team has brought together the herpes virus and a molecule known as Sleeping Beauty to improve a technology known as gene therapy, which aims to manipulate genes to correct for molecular flaws that cause disease.The work, detailed in a paper published online in Gene Therapy, has allowed scientists at the...
Strategy could have implications for multiple tumor typesResearchers at Nationwide Children's Hospital and Johns Hopkins University have discovered that delivering a small molecule that is highly expressed in normal tissues but lost in diseased cells can result in tumor suppression.MicroRNAs (miRNA) are a class of small RNA molecules that are highly expressed in normal tissues and are critical in gene expression and in maintaining normal cell development and cell balance. Dysfunction of...
A new study suggests that delivering small RNAs, known as microRNAs, to cancer cells could help to stop the disease in its tracks. microRNAs control gene expression and are commonly lost in cancerous tumors. Researchers have shown that replacement of a single microRNA in mice with an extremely aggressive form of liver cancer can be enough to halt their disease, according to a report in the June 12 issue of the journal Cell, a Cell Press publication. They delivered the microRNA to the mice...
Phase 1 Data Demonstrate Safety and Evidence of Biological Activity in 9 of 12 Patients; Phase 2 Study Currently Enrolling in 17 Leading U.S. Medical Centers SAN DIEGO, May 28 /PRNewswire/ -- Celladon Corporation presented today Phase 1 data from the Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID), a First-in-Human Phase 1/2 Clinical Trial, in a scientific symposium at the 12th Annual American Society of Gene Therapy Meeting. The Phase 1...
U.S. scientists have turned a relatively benign virus into an infectious form that, in its first gene therapy test, cured cystic fibrosis tissue in culture. Researchers from the University of California-Berkeley and the University of Iowa said their achievement with the benign adeno-associated virus overcomes a major problem of earlier virus-based gene therapy for cystic fibrosis, setting the stage for tests in advanced animal models of the disease. I think it is worthwhile thinking about...
Researchers from the University of California, Berkeley, and the University of Iowa have turned a relatively benign virus into a highly infectious form that is ideal as a carrier for gene therapy.In its first gene therapy test, it completely cured human cystic fibrosis lung tissue in culture.This success with the benign adeno-associated virus (AAV), published this week in the online early edition of the journal Proceedings of the National Academy of Sciences, overcomes a major problem of...
Targeted Genetics Corporation (NASDAQ: TGEN) will report its third quarter 2008 financial results after the close of the financial markets on Wednesday, November 5, 2008. Following the announcement, management will host a conference call and webcast discussion of the financial results and provide a general corporate update beginning at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. You may access the live webcast via the "Events" section found on the homepage of the Company's website at:...
Targeted Genetics Corporation (NASDAQ: TGEN) announced positive results of its now completed Phase 1/2 clinical study with the last subject having finished the protocol-required follow-up. The data demonstrated that tgAAC94, an investigational agent designed to inhibit activity of tumor necrosis factor-alpha (TNF-alpha), a key mediator of inflammation, is well tolerated and may improve disease symptoms in inflammatory arthritis patients. The results of the study were presented yesterday by...
AMSTERDAM, The Netherlands, September 18 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it obtained a license from Amgen to use their GDNF gene for the development of a gene therapy treatment for Parkinson's disease. The combination of this gene with AMT's proprietary adeno-associated virus (AAV) gene therapy platform could potentially allow the development of an effective, long-term treatment for...
