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

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

Research output: Contribution to journalArticlepeer-review

Original languageEnglish
Pages (from-to)67-78
Number of pages12
JournalMolecular therapy. Nucleic acids
Early online date18 Feb 2021
Publication statusPublished - 4 Jun 2021

Cite this