Hoggatt J. 2016. Gene Therapy for "Bubble Boy" Disease. Cell. 166(2):263. Pubmed: 27419862


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.
Copyright © 2016 Elsevier Inc. All rights reserved.

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Jon Hoggatt researches tissue regeneration and stem cell biology, with a particular focus on translational research to enhance bone marrow transplantation.

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