Gene Therapy for "Bubble Boy" Disease - Cell. 2016 Jul 14

Eicosanoids / G-CSF / Mobilization / Transplantation

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.

Author

Hoggatt J

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