Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency

被引：64
|
作者：
Chinen, Javier
Davis, Joie
De Ravin, Suk See
Hay, Beverly N.
Hsu, Amy P.
Linton, Gilda F.
Naumann, Nora
Nomicos, Effie Y. H.
Silvin, Christopher
Ulrick, Jean
Whiting-Theobald, Narda L.
Malech, Harry L.
Puck, Jennifer M.
机构：
[1] NIAID, Host Def Lab, NIH, Dept Hlth & Human Serv, Bethesda, MD 20892 USA
[2] NHGRI, Genet & Mol Biol Branch, NIAID, NIH,DHHS, Bethesda, MD 20892 USA
来源：
BLOOD | 2007年 / 110卷 / 01期
关键词：
BONE-MARROW-TRANSPLANTATION; CHRONIC GRANULOMATOUS-DISEASE; STEM-CELL TRANSPLANTATION; T-CELL; CD34(+) CELLS; TRANSDUCTION; SCID-X1; SURVIVAL; MODEL; IL2RG;
D O I：
10.1182/blood-2006-11-058933
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Retroviral gene therapy can restore immunity to infants with X-linked severe combined immunodeficiency (XSCID) caused by mutations in the IL2RG gene encoding the common gamma chain (gamma c) of receptors for interleukins 2 (IL-2), -4, -7, -9, -15, and -21. We investigated the safety and efficacy of gene therapy as salvage treatment for older XSCID children with inadequate immune reconstitution despite prior bone marrow transplant from a parent. Subjects received retrovirus-transduced autologous peripherally mobilized CD34(+) hematopoletic cells. T-cell function significantly improved in the youngest subject (age 10 years), and multilineage retroviral marking occurred in all 3 children.
引用
收藏
页码：67 / 73
页数：7
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