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: 28 November 2013, source: Science Alert
Every now and again you might read about gene therapy and efforts to correct serious genetic diseases. But I’m betting that very few readers have had gene therapy, nor have they ever met anyone who has, nor will they ever meet that many. The reasons are simple – while these procedures are possible in theory, in practice, it has proved remarkably difficult to insert new genetic materials into human patients and ensure that the new genes are sustainably expressed. The difficulties encountered highlight the fact that we do not yet have enough fundamental knowledge about gene transfer and control. And what’s more, gene therapy is hugely expensive.

Posted on: 27 November 2013, source: ModVive.com
Inside (almost) all of our cells are 23 pairs of chromosomes, long strands of DNA that are packaged tightly together. Each chromosome houses hundreds of genes, each of which is the blueprint for at least one protein. Little messengers called mRNA make copies of the gene blueprints, edits them and carry them to “builders” called ribosomes. The ribosomes read the copy and translate it into long chains of amino acids that are then folded into unique shapes. These globs of amino acids are proteins, that travel throughout our entire body and are in some way vital for everything that happens inside of us. For example, helicase and chromatin are proteins essential to cell division, what allows us to grow and heal, and immunoglobulin, also called antibodies, protect us from infections.

What happens when something goes wrong with our DNA and thus our proteins?

Posted on: 21 November 2013, source: News-Medical
The UK’s first gene therapy trial for advanced heart failure, CUPID 2, has officially begun at the Golden Jubilee National Hospital (GJNH); with the first candidate recently being administered with a dose of the MYDICAR treatment. The GJNH, home of the Scottish National Advanced Heart Failure Service (SNAHFS), announced in April 2013 that it would be participating in the international trial, helping gather a total of 200 patients from 50 institutions worldwide, to determine the effectiveness of the gene therapy treatment, MYDICAR, in advanced heart failure patients.

Posted on: 1 November 2013, source: Boston Globe
Fidelity Biosciences, a venture capital firm that is a subsidiary of Fidelity Investments, and REGENX Biosciences, announced the formation of Dimension Therapeutics, a Cambridge gene therapy company focused on developing treatments for rare diseases such as hemophilia. Dimension has completed an undisclosed Series A financing that was led by Fidelity Biosciences.

Posted on: 2 October 2013, source: NextBigFuture
Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials. Experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have good chance of also getting the first U.S. approval, but the company says it has not yet submitted an application to the FDA.

Posted on: 14 September 2013, source: Science Recorder
Researchers at Oregon Health and Science University (OHSU) have developed a candidate vaccine that appears to completely clear an AIDS-causing virus from the body. Developed at OHSU’s Vaccine and Gene Therapy Unit, the vaccine is now being tested through a non-human primate form of HIV, called simian immunodeficiency virus (SIV). It is hoped that an HIV-form of the vaccine will soon be tested on humans.