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

• Main Authors: Vanessa Strings-Ufombah (Author), Alberto Malerba (Author), Shih-Chu Kao (Author), Sonal Harbaran (Author), Fanny Roth (Author), Ornella Cappellari (Author), Ngoc Lu-Nguyen (Author), Keiko Takahashi (Author), Sophie Mukadam (Author), Georgina Kilfoil (Author), Claudia Kloth (Author), Petrus Roelvink (Author), George Dickson (Author), Capucine Trollet (Author), David Suhy (Author)
• Format: Book
• Published: Elsevier, 2021-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...