Tuesday, April 14, 2009

Gene Therapy Article in TimesOnline

Today's TimesOnline has an interesting analysis piece on the therapeutic potential of gene therapy. Here is an excerpt:

Gene therapies have been a part of medicine for almost two decades. Only recently have they started to deliver on their immense promise after a string of setbacks raised doubts about the safety of manipulating human DNA, centre, to treat disease.

The idea behind gene therapy is that by inserting new copies of a particular gene into human cells, it should be possible to correct defects, or to enhance beneficial biological processes. This can be done with viruses, which introduce their own genetic material into the cells they infect, and can be engineered to carry a human gene.

....Several trials have also started to produce extremely encouraging results. The Anglo-French SCID therapy, which now uses a new vector, seems to be capable of curing the disease indefinitely. Another UCL team, led by Robin Ali, has improved the sight of patients with Leber’s congenital amaurosis, a genetic cause of blindness. Cerepro, an Ark Therapeutics drug that uses the same vector as the foetal growth promoter, has had good results against brain cancer.

....The dream of using gene therapy to treat more systemic diseases such as cystic fibrosis, muscular dystrophy or spinal muscular atrophy, however, remains more distant. The challenge of conveying the replacement gene to cells throughout the body is one that still has to be overcome.