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How to Save the Bubble Boy Story of David Vetter Born on Lived “David, Died in the February plastic Sep.bubble 21,germ-free 1971, 22, boy” 1984 withenvironment SCID SCID Severe Combined Immunodeficiency Gene mutation Absence of functional T cells Defective antibody response Vulnerable to infectious disease Hematopoietic Stem Cell Transplantation Matched transplant Rejection Half-matched transplant Virus infection Graft-versus-host disease Utero transplant Types of Gene Mutation X-linked SCID Adenosine deaminase deficiency(ADA) Purine nucleoside phosphorylase deficiency Omenn syndrome Bare lymphocyte syndrome JAK3 Artemis/DCLRE1C Gene Therapy Disadvantages of Gene Therapy Problems with viral vectors Short-lived nature of cells Immune response Chance of inducing leukemia Summary David Vetter died of SCID in 1971 Reference 1. Friedmann, T.; Roblin, R. (1972). "Gene Therapy for Human Genetic Disease?". Science 175 (4025): 949–955. 2. Sheridan, C. (2011). "Gene therapy finds its niche". Nature Biotechnology 29 (2): 121–128. 3. Woods, N. B.; Bottero, V.; Schmidt, M.; Von Kalle, C.; Verma, I. M. (2006). "Gene therapy: Therapeutic gene causing lymphoma". Nature 440 (7088): 1123. 4. http://en.wikipedia.org/wiki/Somatic_gene_therapy#Problem s 5. http://en.wikipedia.org/wiki/Severe_combined_immunodefici ency
