MIT team reverses a liver disorder in mice by correcting a
mutated gene.
Using a new gene-editing system based on bacterial proteins,
MIT researchers have cured mice of a rare liver disorder caused by a single
genetic mutation.
The findings, described in the March 30 issue of Nature
Biotechnology, offer the first evidence that this gene-editing technique, known
as CRISPR, can reverse disease symptoms in living animals. CRISPR, which offers
an easy way to snip out mutated DNA and replace it with the correct sequence,
holds potential for treating many genetic disorders, according to the research
team.