Glycosaminoglycans as Biomarkers for Mucopolysaccharidoses and Other Disorders

por Amendum, Paige C., Khan, Shaukat, Yamaguchi, Seiji, Kobayashi, Hironori, Ago, Yasuhiko, Suzuki, Yasuyuki, Celik, Betul, Rintz, Estera, Hossain, Jobayer, Xiao, Wendi, Tomatsu, Shunji
Publicado 2021

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