T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years

Science. 1995 Oct 20;270(5235):475-80. doi: 10.1126/science.270.5235.475.

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.

Publication types

  • Clinical Trial

MeSH terms

  • Adenosine Deaminase / administration & dosage
  • Adenosine Deaminase / blood
  • Adenosine Deaminase / deficiency*
  • Adenosine Deaminase / genetics*
  • Adenosine Deaminase / therapeutic use
  • Antibody Formation
  • Base Sequence
  • Child
  • Child, Preschool
  • Female
  • Follow-Up Studies
  • Gene Expression
  • Gene Transfer Techniques*
  • Genetic Therapy*
  • Genetic Vectors
  • Humans
  • Immunity, Cellular
  • Lymphocyte Count
  • Lymphocyte Transfusion
  • Lymphocytes / enzymology
  • Molecular Sequence Data
  • Severe Combined Immunodeficiency / enzymology
  • Severe Combined Immunodeficiency / immunology
  • Severe Combined Immunodeficiency / therapy*
  • T-Lymphocytes* / enzymology
  • T-Lymphocytes* / immunology

Substances

  • Adenosine Deaminase
  • pegademase bovine