Gene therapy for severe combined immunodeficiency.

doi:10.1146/annurev.med.56.090203.104142

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Article

Gene therapy for severe combined immunodeficiency.

Annual review of medicine - 1 Jan 2005

Cavazzana-Calvo Marina, Lagresle Chantal, Hacein-Bey-Abina Salima, Fischer Alain

Abstract excerpt

Studies of severe combined immunodeficiency (SCID), a group of rare monogenic disorders, have provided key findings about the physiology of immune system development. The common characteristic of these diseases is the occurrence of a block in T cell differentiation, always associated with a direct or indirect impairment of B cell immunity. The resulting combined immunodeficiency is responsible for the clinical...

Topics

B-Lymphocytes
DNA Mutational Analysis
Genetic Therapy
Hematopoietic Stem Cell Transplantation
Humans
Infant
Lymphocyte Activation
Phenotype
Severe Combined Immunodeficiency
T-Lymphocytes
Transplantation, Homologous

Source

Open in PubMed

Identifiers

PMID: 15660528
DOI: 10.1146/annurev.med.56.090203.104142

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