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PLoS Genet. 2006 Sep 29;2(9):e133.

A look to future directions in gene therapy research for monogenic diseases.

Author information

1
matthew.porteus@utsouthwestern.edu

Abstract

The concept of gene therapy has long appealed to biomedical researchers and clinicians because it promised to treat certain diseases at their origins. In the last several years, there have been several trials in which patients have benefited from gene therapy protocols. This progress, however, has revealed important problems, including the problem of insertional oncogenesis. In this review, which focuses on monogenic diseases, we discuss the problem of insertional oncogenesis and identify areas for future research, such as developing more quantitative assays for risk and efficacy, and ways of minimizing the genotoxic effects of gene therapy protocols, which will be important if gene therapy is to fulfill its conceptual promise.

PMID:
17009872
PMCID:
PMC1584267
DOI:
10.1371/journal.pgen.0020133
[Indexed for MEDLINE]
Free PMC Article
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