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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Detalles Bibliográficos
Autores principales: Strings-Ufombah, Vanessa, Malerba, Alberto, Kao, Shih-Chu, Harbaran, Sonal, Roth, Fanny, Cappellari, Ornella, Lu-Nguyen, Ngoc, Takahashi, Keiko, Mukadam, Sophie, Kilfoil, Georgina, Kloth, Claudia, Roelvink, Petrus, Dickson, George, Trollet, Capucine, Suhy, David
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7940701/
https://www.ncbi.nlm.nih.gov/pubmed/33738139
http://dx.doi.org/10.1016/j.omtn.2021.02.017

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