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Nanotechnology Now – Press Release: ‘CRISPR-Gold’ fixes Duchenne muscular dystrophy mutation in mice

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Home > Press > ‘CRISPR-Gold’ fixes Duchenne muscular dystrophy mutation in mice.

Abstract:Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease.

A new study shows that a single injection of CRISPR-Gold, as the new delivery system is called, into mice with Duchenne muscular dystrophy led to an 18-times-higher correction rate and a two-fold increase in a strength and agility test compared to control groups.

Through a series of cellular mechanisms, CRISPR-Gold is released into the cells’ cytoplasm and breaks apart, rapidly releasing Cas9 and donor DNA. A single injection of CRISPR-Gold into muscle tissue of mice that model Duchenne muscular dystrophy restored 5.4 percent of the dystrophin gene, which causes the disease, to the wild- type, or normal, sequence.

CRISPR-Gold was also able to reduce tissue fibrosis – the hallmark of diseases where muscles do not function properly – and enhanced strength and agility in mice with Duchenne muscular dystrophy.

When the research team tested CRISPR-Gold’s gene-editing capability in mice, they found that CRISPR-Gold efficiently corrected the DNA mutation that causes Duchenne muscular dystrophy, with minimal collateral DNA damage.

The researchers quantified CRISPR-Gold’s off-target DNA damage and found damage levels similar to that of a typical DNA sequencing error in a typical cell that was not exposed to CRISPR. To test for possible immunogenicity, the blood stream cytokine profiles of mice were analyzed 24 hours and two weeks after the CRISPR-Gold injection.


Article originally posted at

Post Author: Ethan Siegel

1 thought on “Nanotechnology Now – Press Release: ‘CRISPR-Gold’ fixes Duchenne muscular dystrophy mutation in mice

    Preecha Siriprasertkul

    (January 24, 2018 - 12:10 am)

    goodbye diversity

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