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Gene Therapy for ADA-SCID Still Benefitting Patients after Two to Three Years May 11, 2021 “I remember asking the doctor if my daughter was going to die,” said Chelsea Oakley, mother of Cora Oakley, a newborn who was diagnosed with ADA-SCID when she was just seven days old, in April 2017. “His response was, ‘I hope not.’ It was the darkest day of my life.” Cora was the last child to enroll in a clinical trial of a gene therapy for ADA-SCID, or severe combined immunodeficiency due to adenosine deaminase deficiency. After receiving her own corrected cells in September 2017, Cora spent a month in the bone marrow transplant unit close to her family’s home. Observing the other young patients, the Oakleys witnessed what her daughter would have gone through if the experimental gene therapy hadn’t been an option. ....
Credit: Ann Johansson/UCLA Broad Stem Cell Research Center 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 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. ....
Experimental gene therapy successfully treats 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 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. ....