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• W2143729842 endingPage "672" @default.
• W2143729842 startingPage "669" @default.
• W2143729842 abstract "Severe combined immunodeficiency-X1 (SCID-X1) is an X-linked inherited disorder characterized by an early block in T and natural killer (NK) lymphocyte differentiation. This block is caused by mutations of the gene encoding the gammac cytokine receptor subunit of interleukin-2, -4, -7, -9, and -15 receptors, which participates in the delivery of growth, survival, and differentiation signals to early lymphoid progenitors. After preclinical studies, a gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gammac Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells. After a 10-month follow-up period, gammac transgene-expressing T and NK cells were detected in two patients. T, B, and NK cell counts and function, including antigen-specific responses, were comparable to those of age-matched controls. Thus, gene therapy was able to provide full correction of disease phenotype and, hence, clinical benefit." @default.
• W2143729842 created "2016-06-24" @default.
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• W2143729842 date "2000-04-28" @default.
• W2143729842 modified "2023-10-10" @default.
• W2143729842 title "Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease" @default.
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• W2143729842 doi "https://doi.org/10.1126/science.288.5466.669" @default.
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