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Right arrow Genetics, Birth Defects

AAP Grand Rounds 4:24 (2000)
© 2000 American Academy of Pediatrics

GENETICS/BIRTH DEFECTS

Gene Therapy for SCID

Source: Cavazzana-Calvo M, Hacein-Bey S, de Saint Bastile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000;288:669–672.

The first 20% of the full text of this article appears below.

Severe Combined Immunodeficiency (SCID)-X1 Disease is a lethal X-linked recessive disorder in which mutations of the gene coding for a subunit of interleukin receptors result in a block in differentiation of T and natural killer (NK) lymphocytes. SCID-X1 is a reliable model for gene therapy because it is lethal and frequently can be cured by allogenic . . . [Full Text of this Article]

Lawrence R. Shapiro, MD, FAAP
Westchester County Medical Center, Valhalla, NY