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SCIENCE BREAKTHROUGH in gene therapy ... Earlier this month, doctors reported they had successfully treated the first children in the U.S. for severe combined immune deficiency or SCID, sometimes known as “bubble boy” disease. Like Usher, SCID is caused by a mutated recessive gene, meaning children must inherit a defective copy from each parent. And like the Usher trial, the SCID trial included a brother and sister – Colton and Abbygail Ainslie.
ReplyDeleteIn August 2011, doctors reported that two patients with a form of cancer called chronic lymphocytic leukemia had gone into remission after a type of gene therapy treatment that programmed immune system cells to hunt down and kill the leukemia cells.
The idea behind gene therapy sounds straightforward – if a disease is caused by a single faulty gene, then just swap it out for a healthy version. Especially for diseases that affect just one form of tissue, it shouldn’t be too hard. But it isn’t so easy to get the fresh DNA into cells and there is no guarantee that the patient’s cells will accept the new gene and use it.