US EditionGene Therapy Delivers Lasting Immune Protection in Children with Rare Disorder
The gene therapy maintained stable immune function in 59 children with ADA-SCID, with 474 patient-years of follow-up and no treatment-limiting complications reported.
10 Articles
10 Articles
Eliana Nachem was three months old when she was diagnosed with severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, a rare and life-threatening genetic disease. Since that day, Eliana lived in complete medical isolation. No pets, no contact with the outside, with air filters HEPA running constantly and all food and toys sterilized. She was a 'bubble girl'. Conventional therapy for these people is bone marrow trans…
For children who suffer from it, everyday activities such as going to school or playing with their friends can cause dangerous infections that put their life at riskHemeroteca - “Why do I have to be like this?” The documentary that puts in front of the mirror the inequality in the heel test Severe combined immunodeficiency due to ADA enzyme deficiency is a rare disease that, if not treated, can be fatal in the first two years of life. Now, a sci…
The new method uses stem cells of the patient himself, to which a healthy copy of the ADA gene is inserted into the laboratory. Then, these repaired cells are reintroduced into the body and restore the immune system from scratch. Read
Gene therapy delivers lasting immune protection in children with rare disorder
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained immune system function in 59 of 62 children born with ADA-SCID, a rare and deadly genetic immune disorder.
Gene Therapy Provides Long-Term Immune Protection for Children with Rare
An innovative gene therapy developed collaboratively by UCLA, University College London, and Great Ormond Street Hospital has demonstrated remarkable success in restoring durable immune function in children afflicted with adenosine deaminase severe combined immunodeficiency (ADA-SCID), a life-threatening genetic immune disorder. This experimental treatment stands as a groundbreaking advancement in medical science, redefining therapeutic strategi…
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