gene therapy

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

 Waehler R, Russell SJ, Curiel DT. Engineering targeted viral vectors for gene therapy. Nat Rev Genet. 2007 Jul 3; PMID: 17607305

 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

 Kohn DB, Sadelain M, Glorioso JC. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer. 2003 Jul;3(7):477-88. PMID: 12835668

 Prchal JT. Delivery on demand—a new era of gene therapy? N Engl J Med. 2003 Mar 27;348(13):1282-3. PMID: 12660394

 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