Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia

por Kang, Hye-Ri, Waskowicz, Lauren, Seifts, Andrea M., Landau, Dustin J., Young, Sarah P., Koeberl, Dwight D.
Publicado 2019

Hepatic mitochondrial dysfunction is a feature of Glycogen Storage Disease Type Ia (GSDIa)

por Farah, Benjamin L., Sinha, Rohit A., Wu, Yajun, Singh, Brijesh K., Lim, Andrea, Hirayama, Masahiro, Landau, Dustin J., Bay, Boon Huat, Koeberl, Dwight D., Yen, Paul M.
Publicado 2017