Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.

 

Posted on: 1 July 2014, source: European Research Media Center
New virus serotypes are safely used as ‘DNA transporters’ to successfully deliver genes to deficient cells. Between 30 and 40 million people in Europe suffer from rare diseases—many of them children. As most of these diseases have genetic origins, gene therapy is a major hope for their future cure. Until now, however, there have been very few successful trials. Now, the EU-funded project AIPGENE, due to be completed in 2014, may have made significant progress in a gene therapy approach.
The project focussed on the genetic liver disorder, Acute Intermittent Porphyria (AIP). Through an early stage clinical trial, in phase I, it demonstrated the viability of a new approach, based on a so-called, adeno-associated vector (AAV). This is a ‘DNA transporter’ derived from a type of virus and carries the therapeutic gene to liver cells, known as hepatocytes.

Posted on: 26 May 2014, source: NIH
In a milestone for the field of gene therapy, the National Institutes of Health (NIH) will no longer subject all proposed gene therapy clinical trials to review by a special federal advisory committee. “Given the progress in the field, I am confident that the existing regulatory authorities can effectively review most gene transfer protocols and that a streamlined process will reduce duplication and delays in getting gene transfer trials initiated,” said NIH Director Francis Collins in a statement today. Instead, the 40-year-old Recombinant DNA Advisory Committee (RAC) will review only a few trials that pose special risks.

Posted on: 14 May 2014, source: MetroNews
An injection that promises to end obesity seems like the type of claim found only on obnoxious flashing web ads, but it’s entirely plausible that one day we will be able to treat this common problem with just the prick of a needle, according to Jason Dyck, a researcher at the University of Alberta. Two years ago, Dyck and his colleagues published a paper in the journal Nutrition and Diabetes that concluded an injectable adiponectin gene therapy reduced fat and improved insulin sensitivity in mice, despite the fact the test animals were being fed a high-fat diet.

Posted on: 11 April 2014, source: Bio-ITWorld
Celladon today became the first company to receive breakthrough status for a gene therapy treatment in development. The treatment, MYDICAR, is intended to reduce the risk of heart failure in patients with a deficiency of the enzyme SERCA2a. The FDA made its decision to grant breakthrough status to MYDICAR on the basis of Celladon's Phase 1 trial of 39 systolic dysfunction patients. The study reported that subjects receiving high doses of MYDICAR experienced fewer heart failure events than subjects given a placebo, by a factor of more than 80%, and that this reduction was sustained for three years after treatment. No safety issues were reported in this initial trial. Breakthrough status, which is reserved for treatments targeting life-threatening diseases that show significant improvements over the standard of care, allows closer communication with the FDA and potentially an accelerated approval process.

Posted on: 20 March 2014, source: HealthCanal
Physicians and scientists from Munich and Heidelberg have now proven the long-term effectiveness of stem cell gene therapy, based on a study of patients from the first clinical trial worldwide using gene therapy to treat Wiskott-Aldrich syndrome. Yet several years after the therapy, the researchers also observed an increased incidence of acute leukemia among the patients.

Posted on: 13 March 2014, source: International Gene Therapy Consortium
Established at the meeting of the American Society of Gene and Cell Therapy (ASCGT) in May 2013, the Consortium, which is comprised of leading scientists and researchers from more than 50 locations worldwide, has created a website where patients can interact with scientists from the organization. An online course of gene therapy was created with the collaboration of many researchers to facilitate the teaching of this discipline to students in science and medicine. Patients can also ask questions about various genetic diseases. There will also be a virtual meeting place and a forum for discussion on their diseases.