Mice cured of rare liver disorder using new gene-editing system

MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation by using a new gene-editing system based on bacterial proteins.

The findings 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.

"What's exciting about this approach is that we can actually correct a defective gene in a living adult animal," Daniel Anderson, the Samuel A. Goldblith Associate Professor of Chemical Engineering at MIT, a member of the Koch Institute for Integrative Cancer Research, and the senior author of the paper said.

The recently developed CRISPR system relies on cellular machinery that bacteria use to defend themselves from viral infection.

Researchers have copied this cellular system to create gene-editing complexes that include a DNA-cutting enzyme called Cas9 bound to a short RNA guide strand that is programmed to bind to a specific genome sequence, telling Cas9 where to make its cut.

At the same time, the researchers also deliver a DNA template strand. When the cell repairs the damage produced by Cas9, it copies from the template, introducing new genetic material into the genome.

Scientists envision that this kind of genome editing could one day help treat diseases such as hemophilia, Huntington's disease, and others that are caused by single mutations.

The findings are published in the journal Nature Biotechnology.