期刊
SCIENCE
卷 288, 期 5466, 页码 669-672出版社
AMER ASSOC ADVANCEMENT SCIENCE
DOI: 10.1126/science.288.5466.669
关键词
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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 gamma c 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 gamma c Moloney retrovirus-derived Vector and ex vivo infection of CD34(+) cells. After a 10-month follow-up period, gamma c 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.
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