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: 17 February 2014, source: University of Westminster
Researchers at the University of Westminster have developed a groundbreaking method which can be used to test a new innovative cure for hepatitis C, a liver disease caused by the hepatitis C virus (HCV). The cure is the first of its kind ever to be tested in humans and comes in the form of a drug based on gene therapy which is under development by the Australian company Benitec Biopharma. Around 150 million people worldwide are infected with hepatitis C, and more than 350,000 people die every year from hepatitis C related liver diseases. Hepatitis C is one of the leading causes of liver cirrhosis and cancer, and one of the most common and seriously infectious conditions in the world.

Posted on: 29 January 2014, source: WorldBulletin
U.S. researchers have developed tiny nanoparticle robots that can travel through a patient's blood and into tumors where they deliver a therapy that turns off an important cancer gene. The finding, reported in the journal Nature on Sunday, offers early proof that a new treatment approach called RNA interference or RNAi might work in people.

Posted on: 14 January 2014, source: BioNews
Patients in a clinical trial to treat Parkinson's disease with a form of gene therapy have showed signs of significant improvements in their motor-function, according to a report published in the Lancet. Fifteen advanced-stage Parkinson's patients (three from the UK and 12 from France) were followed up a year after being injected with low, mid and high doses of a modified virus containing genes required for brain cells to produce dopamine, as part of the phase I/II study. A lack of dopamine causes patients with Parkinson's to experience tremors and difficulty in coordinating their movement. The researchers observed that after receiving the treatment the patients' scores on movement tests improved on average by 30 percent. The patients also reported having a better quality of life. Although the patients showed some side effects, overall the treatment was found to be safe and no serious side effects were observed.

Posted on: 23 December 2013, source: JapanNews
In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients’ blood cells into soldiers that seek and destroy cancer. A few patients with one type of leukemia were given this one-time, experimental therapy several years ago and some remain cancer-free today. Now, at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, with stunning results.

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?