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

In: Molecular therapy. Nucleic acids, Vol. 24, 04.06.2021, p. 67-78.

Research output: Contribution to journalArticlepeer-review

  • 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
Original languageEnglish
Pages (from-to)67-78
Number of pages12
JournalMolecular therapy. Nucleic acids
Early online date18 Feb 2021
Publication statusPublished - 4 Jun 2021
This open access research output is licenced under a Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Unported License.

ID: 41832607