PABPN1 gene therapy for oculopharyngeal muscular dystrophy

Oculopharyngeal muscular dystrophy is caused by trinucleotide repeat expansions in thePABPN1gene. Here the authors use AAV-based gene therapy to knockdown the mutant gene and replace it with a wild-type allele, and show effectiveness in mice and in patient cells.

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Bibliographic Details
Main Authors:	A. Malerba, P. Klein, H. Bachtarzi, S. A. Jarmin, G. Cordova, A. Ferry, V. Strings, M. Polay Espinoza, K. Mamchaoui, S. C. Blumen, J. Lacau St Guily, V. Mouly, M. Graham, G. Butler-Browne, D. A. Suhy, C. Trollet, G. Dickson
Format:	article
Language:	EN
Published:	Nature Portfolio 2017
Subjects:	Science Q
Online Access:	https://doaj.org/article/3641f54286a54c1ab1a1d2021d381ebf
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PABPN1 gene therapy for oculopharyngeal muscular dystrophy

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