Publication | Open Access
Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease
Raygene Martier,
Marina Sogorb-González,
Janice Stricker-Shaver,
Jeannette Hübener‐Schmid,
Sonay Keskin,
Lodewijk J.A. Toonen,
Štefan Juhás,
Jana Juhásová,
Zdeňka Ellederová,
Jan Motlík,
Molecular Therapy — Methods & Clinical Development