Abstract
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.
MeSH terms
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Adenosine Deaminase / administration & dosage
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Adenosine Deaminase / blood
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Adenosine Deaminase / deficiency*
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Adenosine Deaminase / genetics*
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Adenosine Deaminase / therapeutic use
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Antibody Formation
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Base Sequence
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Child
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Child, Preschool
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Female
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Follow-Up Studies
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Gene Expression
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Gene Transfer Techniques*
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Genetic Therapy*
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Genetic Vectors
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Humans
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Immunity, Cellular
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Lymphocyte Count
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Lymphocyte Transfusion
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Lymphocytes / enzymology
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Molecular Sequence Data
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Severe Combined Immunodeficiency / enzymology
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Severe Combined Immunodeficiency / immunology
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Severe Combined Immunodeficiency / therapy*
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T-Lymphocytes* / enzymology
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T-Lymphocytes* / immunology
Substances
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Adenosine Deaminase
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pegademase bovine