CRISPR Technique Refined for Potential Application in Muscular Dystrophy

Techonology Networks | January 09, 2019

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, Ph.D., 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).
Children with DMD have a gene mutation that interrupts the production of a protein known as dystrophin. Without dystrophin, muscle cells become weaker and eventually die. Many children lose the ability to walk, and muscles essential for breathing and heart function ultimately stop working.

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High-quality and reliable biotech manufacturing is every bit as important as scientific innovation. The significant investment Amgen has made in state-of-the-art manufacturing facilities, robust processes,1,2 and an experienced and engaged workforce reflects our unwavering commitment to excellence in biotech manufacturing.

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