BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

Oculopharyngeal muscular dystrophy (OPMD) is a rare autosomal dominant disease that results from an alanine expansion in the N-terminal domain of Poly-A Binding Protein Nuclear-1 (PABPN1). We have recently demonstrated that a two-vector gene therapy strategy significantly ameliorated the pathology i...

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Bibliographic Details
Main Authors: Vanessa Strings-Ufombah, Alberto Malerba, Shih-Chu Kao, Sonal Harbaran, Fanny Roth, Ornella Cappellari, Ngoc Lu-Nguyen, Keiko Takahashi, Sophie Mukadam, Georgina Kilfoil, Claudia Kloth, Petrus Roelvink, George Dickson, Capucine Trollet, David Suhy
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
oculopharyngeal muscular dystrophy
OPMD
gene therapy
AAV
silence and replace
PABPN1
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121000524

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http://www.sciencedirect.com/science/article/pii/S2162253121000524

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