News FocusGene Therapy

Repair Kits for Faulty Genes

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Science  23 Nov 2001:
Vol. 294, Issue 5547, pp. 1639
DOI: 10.1126/science.294.5547.1639

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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.