Gene therapy offers potential cure to children born without an immune system

An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves them without an immune system.Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is caused by mutations in theADA gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system.For children with the condition, even day-to-day activities like going to school or playing with friends can lead to dangerous, life-threatening infections. If untreated, ADA-SCID can be fatal within the first two years of life.The investigational gene therapy method involves first collecting some of the child ’s blood-forming stem cells, which have the potential to create all types of blood and immune cells. Next, using an approach developed by the research team, a new copy of the ADA gene is delivered into the stem cells by a modified lentivirus, or “viral vector.” The corrected cells are then ret urned to the child’s body, where they are intended to produce a continual supply of healthy immune cells capable of fighting infection.In a study  published todayin the New England Journal of Medicine, co-lead authors Dr. Donald Kohn of UCLA and Dr. Claire Booth of Great Ormond Street Hospital, or GOSH, report two- and three-year outcomes for children treated with the investigational lentiviral gene therap...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news