GM1 Gangliosidosis: Mechanisms and Management

por Rha, Allisandra K, Maguire, Anne S, Martin, Douglas R
Publicado 2021

Base editing corrects the common Salla disease SLC17A5 c.115C>T variant

por Harb, Jerry F., Christensen, Chloe L., Kan, Shih-Hsin, Rha, Allisandra K., Andrade-Heckman, Perla, Pollard, Laura, Steet, Richard, Huang, Jeffrey Y., Wang, Raymond Y.
Publicado 2023