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Gene therapy successfully treats rare immunodeficiency in paediatric patients

An experimental gene therapy developed by researchers at the US’s University of California – Los Angeles (UCLA) and UK’s Great Ormond Street Hospital (GOSH) in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder called that leaves them without an immune system. This condition is called severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).

ADA-SCID is estimated to occur in roughly one in 200,000 to one million newborns worldwide. It is caused by mutations in the ADA gene that impair the activity of the adenosine deaminase enzyme, impairing their immune system and rendering them highly susceptible to severe infections. If untreated, the disease is usually fatal within the first two years of life.

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