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...

Täydet tiedot

Bibliografiset tiedot

Päätekijät:	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
Aineistotyyppi:	Artigo
Kieli:	Inglês
Julkaistu:	Elsevier 2021-06-01
Sarja:	Molecular Therapy: Nucleic Acids
Aiheet:	oculopharyngeal muscular dystrophy OPMD gene therapy AAV silence and replace PABPN1
Linkit:	http://www.sciencedirect.com/science/article/pii/S2162253121000524
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