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Published on: May 27, 2011
Gene Therapy for Inborn Errors of Immunity: Severe Combined Immunodeficiencies
Kritika Chetty1, Ben C Houghton2, Claire Booth3
1Department of Paediatric Immunology and Gene Therapy, Level 3, Zayed Centre for Research Great Ormond Street Hospital, Great Ormond Street, London, WC1N 3JH, United Kingdom.
Abstract:
Severe combined immune deficiency (SCID) causes profound deficiency in T cells and variable deficiencies in B and NK cells. Untreated, the condition is fatal within the first 2 years of life. HSCT has traditionally been the only curative approach; however, success rates are suboptimal in those lacking an HLA-matched donor and conditioning regimens can cause significant toxicity. Gene therapy was pioneered for adenosine deaminase (ADA-SCID) over 3 decades ago and has produced highly successful results. Encouraging data for X-SCID and preclinical work for Artemis-SCID and RAG1-SCID are paving the way for the therapy to become a viable curative treatment option.
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