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RNA-Based Therapy Utilizing Oculopharyngeal Muscular Dystrophy Transcript Knockdown and Replacement

Oculopharyngeal muscular dystrophy (OPMD) is caused by a small expansion of a short polyalanine (polyAla) tract in the poly(A)-binding protein nuclear 1 protein (PABPN1). Despite the monogenic nature of OPMD, no treatment is currently available. Here we report an RNA replacement strategy that has th...

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Main Authors: Aida Abu-Baker (Author), Nawwaf Kharma (Author), Jonathan Perreault (Author), Alanna Grant (Author), Masoud Shekarabi (Author), Claudia Maios (Author), Michele Dona (Author), Christian Neri (Author), Patrick A. Dion (Author), Alex Parker (Author), Luc Varin (Author), Guy A. Rouleau (Author)
Format: Book
Published: Elsevier, 2019-04-01T00:00:00Z.
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