Research teams use CRISPR to successfully snip parts of muscular dystrophy away

01/05/2016 - 15:32

Jocelyn Kaiser

The red-hot genome editing tool known as CRISPR has scored another achievement: Researchers have used it to treat a severe form of muscular dystrophy in mice. Three groups report today in Science that they wielded CRISPR to snip out part of a defective gene in mice with Duchenne muscular dystrophy (DMD), allowing the animals to make an essential muscle protein.


Ref: In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science (31 December 2015) | DOI: 10.1126/science.aad5143

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science (31 December 2015) | DOI: 10.1126/science.aad5725

In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science (31 December 2015) | DOI: 10.1126/science.aad5177