Evading the AAV Immune Response in Mucopolysaccharidoses

por Piechnik, Matthew, Sawamoto, Kazuki, Ohnishi, Hidenori, Kawamoto, Norio, Ago, Yasuhiko, Tomatsu, Shunji
Publicado 2020

Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management

por Sawamoto, Kazuki, Álvarez González, José Víctor, Piechnik, Matthew, Otero, Francisco J., Couce, Maria L., Suzuki, Yasuyuki, Tomatsu, Shunji
Publicado 2020

Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy

por Piechnik, Matthew, Amendum, Paige C., Sawamoto, Kazuki, Stapleton, Molly, Khan, Shaukat, Fnu, Nidhi, Álvarez, Victor, Pachon, Angelica Maria Herreño, Danos, Olivier, Bruder, Joseph T., Karumuthil-Melethil, Subha, Tomatsu, Shunji
Publicado 2022