Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

doi:10.1126/science.288.5466.669

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Article

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

Science (New York, N.Y.) - 28 Apr 2000

Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova J L, Bousso P, Deist F L, Fischer A

Abstract excerpt

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...

Topics

Antigens, CD34
B-Lymphocytes
Gene Transfer Techniques
Genetic Therapy
Genetic Vectors
Hematopoietic Stem Cell Transplantation
Hematopoietic Stem Cells
Humans
Immunoglobulins
Infant

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Killer Cells, Natural

Lymphocyte Activation

Lymphocyte Count

Moloney murine leukemia virus

Mutation

Receptors, Antigen, T-Cell

Receptors, Interleukin

Severe Combined Immunodeficiency

Source

Open in PubMed

Identifiers

PMID: 10784449
DOI: 10.1126/science.288.5466.669

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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