Call in the backup
Elie Dolgin • November 7, 2011
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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