Promising genetic 'spell check' for spinal muscular atrophy developed
University of Delaware researcher Eric Kmiec is an editor. But instead of taking on 26 letters and the English language, he addresses the 4 letters of the genetic code (AGCT) and the genes they comprise. His latest editing work, in which he substitutes a working gene for a malfunctioning one, offers hope for patients with spinal muscular atrophy, the genetic disease that is the most frequent killer of very young children. Preliminary tests of the technique in mice at The Jackson Laboratory were “very promising,” and there are hopes for human clinical testing in the near future, according to a news release about the research.
