CRISPR Nanoparticles Repair Duchenne Muscular Dystrophy Gene

October 6, 2017

(GEN) – cientists have developed a gold nanoparticle technology for delivering the CRISPR/Cas9 gene-editing system to cells that, when tested in the mdx mouse model of Duchenne muscular dystrophy (DMD), repaired the faulty DMD gene, leading to improved strength and agility and reduced fibrosis. Professor Niren Murthy, Ph.D., the University of California, Berkeley (UC Berkeley) researcher who led development of the CRISPR-Gold platform, suggested to GEN that human clinical gene-editing trials using the system could feasibly start within the next few years.

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