Open AccessCall in the backup
Author(s) -
Elie Dolgin
Publication year - 2012
Publication title -
nature medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.536
H-Index - 547
eISSN - 1546-170X
pISSN - 1078-8956
DOI - 10.1038/nm1112-1602
Subject(s) - backup , reboot , spinal muscular atrophy , genome editing , gene , biology , medicine , genome , genetics , bioinformatics , computer science , database , operating system
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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