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Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency

A new gene therapy is able to safely restore the immune function in children with severe combined immunodeficiency due to adenosine deaminase deficiency, a rare and devastating disease also known by its acronym in English: ADA-SCID.

The validity of the therapy has been proven in research supported by the National Institutes of Health (NIH) in the United States.

The researchers found that 48 of the 50 children who received gene therapy continued to maintain restored immune system function two to three years after treatment, and during this time they did not need additional treatments for their disease.

By some estimates, ADA-SCID affects 1 in 200,000 newborns worldwide. According to other estimates, the proportion is one affected for every million newborns.

ADA-SCID is caused by mutations in the ADA gene that alter the activity of the enzyme adenosine deaminase, necessary for the healthy function of the immune system. This alteration makes children with this disease very susceptible to serious infections. If left untreated, the disease is fatal, usually in the first two years of life.

Results on ADA-SCID treatment validity come from three separate phase 1/2 clinical trials, two conducted in the United States and one in the United Kingdom.

In this artistic recreation, a DNA double helix rests on a printed illustration of the letters A, T, C and G of DNA. (Image: Darryl Leja, NHGRI, NIH)

The US clinical trials, led by Dr. Donald Kohn of UCLA (University of California, Los Angeles), involved 30 participants with ADA-SCID ranging in age from 4 months to 4 years.

The British clinical trials, conducted at GOSH (Great Ormond Street Hospital) and led by Claire Booth, involved 20 participants ranging in age from 4 months to 16 years.

The results of the clinical trials are detailed in the academic journal New England Journal of Medicine, under the title “Autologous ex vivo lentiviral gene therapy for adenosine deaminase deficiency”. (Source: NCYT from Amazings)

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