Stunning gene therapy breakthrough driven by great dedication and graft | Robin McKie

We need more than ever to celebrate advances in medical science – though they may take years to emergeThere has been a surprising outbreak of the use of the c-word among medical researchers over the past few days. Normally cautious in their language, they have nevertheless been wielding the term “cure” when discussing the long-term potential of two separate treatments for inherited ailments that were announced last week. Such enthusiasm is striking.In one case, scientists based at St Bartholomew ’s, London – who have been working on the inherited bleeding disorder haemophilia A –outlined how they had used a virus to carry the gene for the blood-clotting chemical, factor VIII (which patients lack) to their livers. Production of the missing chemical was restored and their bleeding halted. The development, according to theWorld Federation of Hemophilia, now points “the way to a cure” for the condition, which affects around 400,000 people worldwide.Continue reading...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Medical research Science Genetics Biology Cystic fibrosis Society Huntington's disease Alzheimer's Dementia Source Type: news