Gene Therapy for Severe Combined Immunodeficiency: Are We There Yet?

Overview

Journal J Clin Invest

Specialty General Medicine

Date 2007 Jun 6

PMID 17549248

Citations 52

Authors

Marina Cavazzana-Calvo

Alain Fischer

Affiliations

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Abstract

Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related HLA-mismatched donor is used and thus represents an attractive alternative. In this review, we summarize the advantages and limitations associated with the use of gene therapy to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed.

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