Researchers at the University of Delaware have discovered a novel technique—that acts like a “spell-checker” for correcting a misspelling in the DNA code—to repair the defective gene that causes spinal muscular atrophy (SMA). This hereditary neuromuscular disease is the number-one genetic killer of children under two years old. Babies born with Type 1 SMA, the most severe form of the disease, can’t walk, crawl, sit unsupported, lift their heads, or breathe normally. Fifty percent die before their second birthday.

The research is published in the Jan. 14 online edition of Experimental Cell Research. The study was supported by $477,500 in National Tobacco Settlement funds to the state of Delaware. The research grant was awarded through the Delaware Health Fund.

“Think of it like a spell-check program—we’re erasing the wrong letter in the DNA code and putting the right one in,” said Eric Kmiec, professor of biological sciences at UD.
Read the full press release