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

R. Michael Blaese, Kenneth W. Culver, A. Dusty Miller, Charles S. Carter, Thomas Fleisher, Mario Clerici, Gene Shearer, Lauren Chang, Yawen Chiang, Paul Tolstoshev, Jay J. Greenblatt, Steven A. Rosenberg, Harvey Klein, Melvin Berger, Craig A. Mullen, W. Jay Ramsey, Linda Muul, Richard A. Morgan, W. French Anderson

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Pages (from-to)475-480
Number of pages6
JournalScience
Volume270
Issue number5235
Publication statusPublished - 1995

ASJC Scopus subject areas

  • General

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