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Nonviral CRISPR-Gold Editing Technique Fixes Duchenne Muscular Dystrophy Mutation in Mice

Accepted submission by takyon at 2017-10-14 14:59:33 from the gene-rich dept.

A new and non-viral approach to CRISPR has been used to treat Duchenne muscular dystrophy in mice []:

A new version of the CRISPR []-Cas9 gene-editing technology called CRISPR-Gold has successfully restored the correct sequence of the dystrophin gene in a mouse model of Duchenne muscular dystrophy (DMD), a new study revealed.

Researchers found that an injection of CRISPR-Gold into DMD mice led to an 18-times-higher correction rate and a two-fold increase in a strength and agility test compared to control groups, according to a press release [].

The study, "Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair []," [DOI: 10.1038/s41551-017-0137-2] [DX []] was published in the journal Nature Biomedical Engineering [].

[...] Unfortunately, methods of delivering the components of this system, which include an RNA molecule called a guide RNA, a protein called the Cas9 nuclease, and the correct DNA sequence to replace the mutation (via donor DNA), have not been fully developed for human use. A primary technique used to deliver the components of this system relies on viruses, but this technique is plagued by complications and unwanted side effects.

In response, researchers at the University of California, Berkeley [] have developed a new approach called CRISPR-Gold, which used gold nanoparticles to deliver the components of this system in a mouse model of DMD. This method works by using gold nanoparticles to coat a modified DNA molecule that binds the donor DNA, which in turn is bound to Cas9 and the guide RNA.

This entire system is then coated by a polymer that will interact with a cell membrane and allow entry into a cell. Then, the components of the system are released into the cell as the coat breaks apart upon entry. The guide RNA, the Cas9 nuclease, and the donor DNA can then make their way into the nucleus and correct the mutation.

Also at TheScientist [].

Previously: FDA Panel Recommends Rejection of Duchenne Muscular Dystrophy Treatment []
Marathon Pharmaceuticals is Part of the Problem []
Marathon Pharmaceuticals Cashes Out on Regulatory Loopholes []
What is a Muscle Protein Doing in the Brain? []

Original Submission