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 ...
Hosted on MSN

Gene Therapy Shows Lasting Benefit for Children With Rare Disorder

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational gene therapy showed sustained clinical efficacy and safety at a median ...
Hosted on MSN

Lentiviral Gene Therapy Shows Sustained Clinical Efficacy for ADA-SCID

FRIDAY, Oct. 17, 2025 (HealthDay News) -- Autologous CD34+ hematopoietic stem-cell lentiviral gene therapy shows sustained clinical efficacy for severe combined immunodeficiency (SCID) due to ...
Nature

Adenosine Deaminase Deficiency and Severe Combined Immunodeficiency

Adenosine deaminase (ADA) deficiency is a rare autosomal recessive disorder that underpins a severe form of combined immunodeficiency (SCID), resulting in the accumulation of toxic metabolites that ...