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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書誌詳細
出版年:Mol Ther Nucleic Acids
主要な著者: Abu-Baker, Aida, Kharma, Nawwaf, Perreault, Jonathan, Grant, Alanna, Shekarabi, Masoud, Maios, Claudia, Dona, Michele, Neri, Christian, Dion, Patrick A., Parker, Alex, Varin, Luc, Rouleau, Guy A.
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Gene & Cell Therapy 2019
主題:
Article
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC6403420/
https://ncbi.nlm.nih.gov/pubmed/30831428
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.02.003
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