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Children suffering from a rare and life-threatening condition have been successfully treated by a gene therapy that makes use of their own stem cells. ....
By City News Service May 12, 2021 LOS ANGELES (CNS) - 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, according to a study published today. Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is caused by mutations in the ADA 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. ....
Gene therapy offers cure for an immune disorder Genetically modified stem cells restore immune function in more than 95% of patients with the rare disease. Dr. Donald Kohn with a member of his lab. Credit: Ann Johansson/UCLA Broad Stem Cell Research Center An international team of scientists have developed an experimental gene therapy treatment for children with a severe immune disorder, and it’s showing promise. As described in a paper published in the New England Journal of Medicine, clinical trials in the US and UK have successfully treated 48 out of 50 participants born with a rare and life-threatening disease known as severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). ....