Gene Therapy for "Bubble Boy" Disease.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 27419862.
- Also identified by DOI 10.1016/j.cell.2016.06.049.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
Adenosine deaminase (ADA) deficiency results in the accumulation of toxic metabolites that destroy the immune system, causing severe combined immunodeficiency (ADA-SCID), often referred to as the "bubble boy" disease. Strimvelis is a European Medicines Agency approved gene therapy for ADA-SCID patients without a suitable bone marrow donor.