She Was Born Without an Immune System. Gene Therapy Saved Her Life.

Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...
New Atlas

95% of kids with “bubble boy” disease cured by one-time gene therapy

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...
Nature

Respiratory viral infections in primary immune deficiencies: significance and relevance to clinical outcome in a single BMT unit

Severe combined immunodeficiency syndrome (SCID) describes a group of inherited diseases characterised by a failure of both cellular and humoural immunity. Affected children present with recurrent ...