Abstract
The successful use of retroviral gene transfer to treat 10 patients with X-linked severe combined immunodeficiency (SCID-X1) has been welcomed as evidence of the therapeutic potential of gene therapy. However, as Williams and Baum suggest in their Perspective, the discovery that 2 of the 10 patients developed leukemia within 3 years of gene therapy (Hacein-Bey-Abina et al.) reinforces the need to develop even more specific gene therapy interventions.
Publication types
-
Comment
-
Research Support, Non-U.S. Gov't
-
Research Support, U.S. Gov't, P.H.S.
MeSH terms
-
Adaptor Proteins, Signal Transducing
-
Clinical Trials as Topic
-
DNA-Binding Proteins / genetics
-
Gene Transfer Techniques
-
Genes, Tumor Suppressor
-
Genetic Therapy* / adverse effects
-
Genetic Vectors
-
Hematopoietic Stem Cell Transplantation
-
Hematopoietic Stem Cells / physiology
-
Humans
-
Infant
-
LIM Domain Proteins
-
Leukemia-Lymphoma, Adult T-Cell / etiology*
-
Metalloproteins / genetics
-
Mutagenesis, Insertional
-
Proto-Oncogene Proteins
-
Proto-Oncogenes
-
Receptors, Interleukin-2 / genetics
-
Retroviridae / genetics
-
Risk Factors
-
Severe Combined Immunodeficiency / therapy*
-
T-Lymphocytes / physiology
-
Transgenes
Substances
-
Adaptor Proteins, Signal Transducing
-
DNA-Binding Proteins
-
LIM Domain Proteins
-
LMO2 protein, human
-
Metalloproteins
-
Proto-Oncogene Proteins
-
Receptors, Interleukin-2