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Researchers Overcome Hurdle In CRISPR Gene Editing For Muscular Dystrophy
Clinical Trials and Research

Researchers Overcome Hurdle in CRISPR Gene Editing for Muscular Dystrophy

The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions. A research team led by Dongsheng Duan, PhD, at the University of Missouri School of Medicine has identified and overcome a barrier in CRISPR gene editing that may lay the foundation for sustained treatments using the technique.

CRISPR gene editing is inspired by the body’s natural defensive ability to fend off viruses. The technology enables researchers to alter DNA sequences by cutting out and replacing a mutation in the genome, which has the potential to treat a variety of genetic diseases and conditions. Duan, along with his collaborators at MU, the National Center for Advancing Translational Sciences at the National Institutes of Health and Duke University, are studying how to harness CRISPR to treat Duchenne muscular dystrophy (DMD).

Jan 8, 2019
Coping With Duchenne: Parents Swap Stories At ‘Futures’ Conference
People

Coping with Duchenne: Parents Swap Stories at ‘Futures’ Conference

Until her son, Will, was diagnosed with Duchenne muscular dystrophy as a 7-year-old, speech pathologist Tiffany Cook had never heard of the disease.

Today, it’s literally her full-time job.

As family resource manager with the nonprofit organization CureDuchenne, Cook — who works from her home in Dallas — helps other moms and dads of newly diagnosed Duchenne boys navigate individualized education programs (IEPs), among other challenges.

Dec 12, 2018