The application of DNA repair vectors to gene therapy

Betsy T. Kren, Clifford J Steer

Research output: Contribution to journalReview articlepeer-review

11 Scopus citations

Abstract

The nature of DNA, the sequence of the human genome and our increased understanding of the genetic basis of many inherited and acquired disorders have made the possibility of curing diseases a reality. The modulation of a host's genome is now the ultimate goal in the treatment of genetic diseases. Historically, gene therapy recognized two very different approaches: gene replacement or augmentation and gene repair. Gene repair precisely targets and corrects the chromosomal mutation responsible for a genetic and/or acquired disorder. Many recent advances have been made in this area of research.

Original languageEnglish (US)
Pages (from-to)473-481
Number of pages9
JournalCurrent opinion in biotechnology
Volume13
Issue number5
DOIs
StatePublished - Oct 1 2002

Bibliographical note

Funding Information:
This work was supported in part by grants PO1-HD32652 (BTK), P01-HL655578 and P01-HL55552 (CJS) from the National Institutes of Health.

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