The most common genetic killer of infants, a disease known as spinal muscular atrophy, is caused by mutations in a single gene. The human genome contains its own backup system—near-identical copies of the defective gene—yet these secondary sequences rarely get used correctly. As Elie Dolgin finds out, drug companies hope to change that, with the first wave of targeted therapies that reboot the body's backup system now entering clinical trials.
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Change history
11 January 2013
The article entitled "Call in the backup" in the November 2012 issue (Nat. Med. 18, 1602–1606, 2012) misspelled Rajesh Ranganathan's first name as Rajan. The error has been corrected in the HTML and PDF versions of the article.
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Dolgin, E. Call in the backup. Nat Med 18, 1602–1605 (2012). https://doi.org/10.1038/nm1112-1602
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DOI: https://doi.org/10.1038/nm1112-1602
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