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gene therapy

Wednesday 21 April 2004

Unlike the multiple mechanisms of endogenous DNA repair, gene repair (or gene correction) refers to a form of gene therapy, which precisely targets and corrects chromosomal mutations responsible for a disorder.

It does so by replacing the flawed DNA sequence with the desired sequence, using techniques such as oligonucleotide-directed mutagenesis.

Genetic mutations requiring repair are normally inherited, but in some cases they can also be induced or acquired (such as in cancer).

References

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- Melo LG, Pachori AS, Gnecchi M, Dzau VJ. Genetic therapies for cardiovascular diseases. Trends Mol Med. 2005 May;11(5):240-50. PMID: #15882612#

- Glover DJ, Lipps HJ, Jans DA. Towards safe, non-viral therapeutic gene expression in humans. Nat Rev Genet. 2005 Apr;6(4):299-310. PMID: #15761468#

- Lichty BD, Power AT, Stojdl DF, Bell JC. Vesicular stomatitis virus: re-inventing the bullet. Trends Mol Med. 2004 May 1;10(5):210-216. PMID: #15121047#

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- Yoon JW, Jun HS. Recent advances in insulin gene therapy for type 1 diabetes. Trends Mol Med. 2002 Feb;8(2):62-8. PMID: #11815271#