Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

RNA interference via the endogenous miRNA pathway regulates gene expression by controlling protein synthesis through post-transcriptional gene silencing. In recent years, miRNA-mediated gene regulation has shown potential for treatment of neurological disorders caused by a toxic gain of function mec...

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Bibliographic Details
Published in:Journal of visualized experiments no. 138
Main Authors: Pourshafie, Naemeh, Lee, Philip R, Chen, Ke-Lian, Harmison, George G, Bott, Laura C, Fischbeck, Kenneth H, Rinaldi, Carlo
Format: Journal Article
Language:English
Published: United States MyJove Corporation 10-08-2018
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