gene therapy archive.

Gene therapy for hearing loss

Hearing loss can be caused by advancing years, or by head injuries or loud noises, all of which lead to loss of the sensory hair cells. A new gene therapy could have potential to replace the damaged hair cells, but isn’t likely to be the answer by itself. The study was published in the Journal of Neuroscience.

Gene therapy reprograms heart scar tissue

Myocardial infarctions (MI or heart attacks) lead to scar tissue that can’t regenerate and doesn’t function as well as normal tissue. Based on in vitro studies showing that using three genes could reprogram the fibroblasts in the scar tissue to create myocytes (heart muscle cells) directly (without going via the stem cell state), researchers from University of California San Francisco believe that gene therapy could turn scar tissue into viable heart muscle cells.

CF gene therapy trial gets go-ahead

Cystic fibrosis (CF) is the commonest lethal inherited disease in the UK, affecting around 9,500 people nationally and over 90,000 worldwide. But there may be hope – a UK gene therapy trial for cystic fibrosis is expected to begin in March, in 130 adults and children. This is the largest …

A magic bullet for haemophilia B

Haemophilia B is an inherited bleeding disorder caused by a mutation to the gene for factor IX, a blood clotting factor. It is X-linked and recessive, so it generally affects males but can be carried silently by females and passed onto their male offspring. Haemophilia B can be life-threatening, shortens lifespan, and makes daily life complicated. Researchers at University College London and St Jude Children’s Research Hospital have created a gene therapy and shown it to be effective in early studies.

Making gene therapy better

Gene therapy has been touted as the brave new world of therapeutics for many years. While it hasn’t changed the world in perhaps the way we thought it would in the early days, gene therapies are gradually making their way through the clinic, and steps are being made to make them safer and more effective and efficient. Research published in the FASEB Journal has combined two types of site-specific recombinases to guide exactly where new genetic material is inserted into a cell’s DNA.

A new cell and gene therapy poll

Wanted: nominations for the top 10 cell therapy & top 10 gene therapy events of 2011. We at Phacilitate are compiling the top 10 events in cell therapy, gene therapy and regenerative medicine from 2011 and need your help.

7th annual North American Cell & Gene Therapy Forum

The programme for Phacilitate’s 7th annual North American Cell & Gene Therapy Forum 2012 is being put together in collaboration with an advisory board and the organisers would like your help too.

Broad-spectrum cancer DNA vaccination shows potential

Prostate cancer is the most common cancer in men. It is generally a slow growing form of cancer, though some men have a more aggressive form of the disease. Animal studies of a prostate cancer DNA vaccine have suggested a new approach to cancer treatment that may have potential to stabilise or cure tumours.

CF gene therapy faces delays

A key UK-based gene therapy trial programme for a treatment for the lung disease cystic fibrosis has been halted because of lack of funding.

Gene therapy and Parkinson’s disease

Parkinson’s disease is a degenerative disorder that affects movement, and is most common in older people. Researchers in the US have just completed the world’s first successful clinical trial of gene therapy in people with Parkinson’s disease.