Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

by Maria Dahl, Emma M.K. Smith, Sarah Warsi, Michael Rothe, Maria J. Ferraz, Johannes M.F.G. Aerts, Azadeh Golipour, Claudia Harper, Richard Pfeifer, Daniella Pizzurro, Axel Schambach, Chris Mason, Stefan Karlsson
Published 2021-03-01