Title: RNA-Based Therapy Utilizing Oculopharyngeal Muscular Dystrophy Transcript Knockdown and Replacement.
Authors: Abu-Baker A, Kharma N, Perreault J, Grant A, Shekarabi M, Maios C, Dona M, Neri C, Dion PA, Parker A, Varin L, Rouleau GA
Journal: Mol Ther Nucleic Acids
Published: 2019-02-15
Volume: 15
Pages: 12-25