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UCLA: Gene Therapy Offers Potential Cure to Kids Born Without Immune System


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. ....

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Gene therapy offers cure for an immune disorder


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).  ....

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