Tag: <span>adeno-associated virus (AAV)</span>

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High-Throughput Method Speeds Discovery of Improved Vectors For Gene Delivery To Diverse Brain Cell Types
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High-Throughput Method Speeds Discovery of Improved Vectors For Gene Delivery To Diverse Brain Cell Types

by Lori Dajose Viruses are nature’s Trojan horses: They gain entrance to cells, smuggle in their genetic material, and use the cell’s own machinery to replicate. For decades, scientists have studied how to minimize their deleterious effects and even repurpose these invaders to deliver not their own viral genome, but therapeutics for treating disease and...

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Gene transfer corrects severe muscle defects in mice with Duchenne muscular dystrophy

Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called dystrophin. Researchers at the University of Missouri, National Center for Advancing Translational Sciences, University of Washington, and Solid Biosciences, LLC, have developed a new gene transfer approach that uses an adeno-associated virus vector...