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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
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Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment
Harry Wilton-Clark1, Toshifumi Yokota1
1University of Alberta, Department of Medical Genetics, Edmonton, AB, Canada.
Med (New York, N.Y.)
|December 9, 2023
Abstract:
Gene therapies, notably those leveraging CRISPR and adeno-associated virus vectors (AAVs), have risen to the forefront of potential treatments for neuromuscular disorders. The recent demise of a Duchenne muscular dystrophy (DMD) patient following a trial utilizing CRISPR transactivation with AAV has cast a spotlight on the potential risks associated with these approaches.1.
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