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Gene Therapy for "Bubble Boy" Disease. 2016

Jonathan Hoggatt
Cancer Center and Center for Transplantation Sciences, Massachusetts General Hospital, Boston, MA 02129, USA. Electronic address: hoggatt.jonathan@mgh.harvard.edu.

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.

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