Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease

by Raygene Martier, Marina Sogorb-Gonzalez, Janice Stricker-Shaver, Jeannette Hübener-Schmid, Sonay Keskin, Jiri Klima, Lodewijk J. Toonen, Stefan Juhas, Jana Juhasova, Zdenka Ellederova, Jan Motlik, Eva Haas, Sander van Deventer, Pavlina Konstantinova, Huu Phuc Nguyen, Melvin M. Evers
Published 2019-12-01