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Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
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Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
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Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
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Journal Article
Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
2010
Overview
In a long-term (up to 11 years) follow-up of nine patients with X-linked severe combined immunodeficiency treated with retroviral insertion of a normal common γ-chain gene into hematopoietic progenitors, seven had durable T-cell reconstitution and long-term survival. B-cell immunity was not corrected. Acute leukemia developed in four patients. In patients without a matched marrow donor, gene therapy may be an option but is associated with serious risks.
The cytokine receptor common γ chain, which is encoded by the interleukin-2 receptor subunit gamma (
IL2RG
) gene, is a critical functional component of the receptors for interleukin-2, interleukin-4, interleukin-7, interleukin-9, interleukin-15, and interleukin-21.
1
Naturally occurring mutations in
IL2RG
are responsible for X-linked severe combined immunodeficiency (SCID-X1) disease. This condition is characterized by the complete lack of T cells and natural killer cells, whereas B cells are present.
2
,
3
Hematopoietic stem-cell transplantation is a lifesaving therapy. Despite associated improvements in the survival rate, however, non−HLA-identical hematopoietic stem-cell transplantation has a number of drawbacks. For example, reconstitution of T-cell function . . .
Publisher
Massachusetts Medical Society
Subject
/ Biological and medical sciences
/ Genetic Therapy - adverse effects
/ Humans
/ Infant
/ Interleukin Receptor Common gamma Subunit - deficiency
/ Interleukin Receptor Common gamma Subunit - genetics
/ Killer Cells, Natural - physiology
/ Leukemia
/ Patients
/ Precursor Cell Lymphoblastic Leukemia-Lymphoma - etiology
/ Severe Combined Immunodeficiency - immunology
