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Science 23 November 2001:
Vol. 294. no. 5547, p. 1639
DOI: 10.1126/science.294.5547.1639
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News Focus

GENE THERAPY:
Repair Kits for Faulty Genes

Dan Ferber

Rather than providing intact genes to make up for the defective version nature provided, a few researchers are developing molecular toolkits to correct mutations in the genome. This strategy could make the difference in treating diseases that require the right amount of therapeutic protein at the right time. Some techniques offer potential means to achieve a longtime dream of gene therapists: a lasting cure for genetic disease.

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Science. ISSN 0036-8075 (print), 1095-9203 (online)