Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency
نویسندگان
چکیده
منابع مشابه
In vitro correction of JAK3-deficient severe combined immunodeficiency by retroviral-mediated gene transduction
Mutations affecting the expression of the Janus family kinase JAK3 were recently shown to be responsible for autosomal recessive severe combined immunodeficiency (SCID). JAK3-deficient patients present with a clinical phenotype virtually indistinguishable from boys affected by X-linked SCID, a disease caused by genetic defects of the common gamma chain (gamma c) that is a shared component of th...
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X-linked severe combined immunodeficiency (XSCID) is a lethal disease caused by a defect in the gene encoding the common gamma chain (gamma-c) of the receptor for interleukin-2 (IL-2), IL-4, IL-7, IL-9, and IL-15. Allogeneic bone marrow transplantation, the current therapy of choice for this defect, is often complicated by graft-versus-host disease and/or incomplete reconstitution of B-lymphocy...
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ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2017
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2016.064