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: 2 September 2010, source: ScienceDaily
German scientists from Tübingen and Mainz have developed a blood test that can reliably detect gene doping even after 56 days. Scientists at the universities in Tübingen and Mainz have developed a test that can provide conclusive proof of gene doping. "For the first time, a direct method is now available that uses conventional blood samples to detect doping via gene transfer and is still effective if the actual doping took place up to 56 days before," Professor Perikles Simon, MD, PhD from Johannes Gutenberg University Mainz, Germany explained. "This represents a relatively low-cost method of detecting several of the most common doping genes," Simon stated in the presentation of the process.

Posted on: 1 September 2010, source: FDA
The FDA will convene a one-day workshop to facilitate an exchange of information about best practices in conducting cell and gene therapy clinical trials in pediatric populations. Cellular and gene therapies are the subject of great interest as novel products that potentially may improve the lives of patients by restoring lost function and modifying the nature and course of diseases. However, these therapies are not without risks: indeed, the novelty of these products contributes to their real and potential risks. Conducting clinical trials with novel products in pediatric patients requires special scrutiny to ensure that the rights of subjects are protected and that potential risks and benefits are appropriately balanced.
The purpose of the workshop is to gather information from Institutional Review Boards (IRBs), gene and cellular therapy clinical researchers, and other stakeholders regarding best practices related to cell and gene therapy clinical trials in pediatric populations, as well as challenges and considerations in the review of these clinical trials.

Posted on: 30 August 2010, source: Tonic.com
By taking a different approach, scientists have developed the next promising cancer treatment: targeted gene therapy. Skin cancer is scary, and it's tricky. If caught early it is easily treatable, but if not, it's potentially deadly. Even the best dermatologists can't spot everything. Targeted gene therapy is the latest treatment recognized by the New England Journal of Medicine. The new approach is proving fruitful when it comes to patients with the deadliest form of skin cancer: metastasized melanoma.

Posted on: 28 August 2010, source: ScienceDaily
Current antiepileptic drugs (AEDs) have many side-effects, among others slowing down brain activity, which in turn reduces patients' ability to react. These side-effects could be eliminated if genes that counteract seizures could be introduced into the brain. Professor Merab Kokaia at Lund University in Sweden has obtained promising results in animal experiments.

Posted on: 20 July 2010, source: CliniGene

Programme title: CliniGene, (Contract number LSHB-CT-2006-018933), the European Network of Excellence for the Advancement of Clinical Gene Transfer and Therapy.

CliniGene seeks additional partners to enter its Joint Programme Activity on the topics:
Topic 1. Non-viral integrating systems for clinical gene therapy and development of human iPS, in particular transposon based technology;
Topic 2. AAV for in vivo gene therapy, in particular mechanisms leading to tight transcription regulation;
Topic 3. Technology transfer to SMEs for: (i) systems which have benefited from common evaluation and fine-tuning inside the NoE; (ii) a technology that is believed to complement the CliniGene current portfolio with view to performing side to side comparisons with gene transfer technologies already evaluated within CliniGene.

Posted on: 8 July 2010, source: The Wall Street Journal
Researchers have launched a new gene-therapy trial for children with a rare disease known as "bubble boy syndrome," reflecting fresh hopes that the strategy of delivering working genes can be used to treat many intractable ailments.
In the new study, sponsored in the U.S. by investigators at Children's Hospital Boston and expected to open at five sites around the world, scientists plan to enroll 20 boys with SCID-X1, which stands for severe combined immunodeficiency, X-linked—a genetic condition that affects boys and leaves them unable to fight germs. Without treatment, which is currently possible only by bone-marrow transplantation, most children die before age one.