Progranulin as a Novel Factor in Gaucher Disease

por Choy, Francis Y.M., Christensen, Chloe L.
Publicado 2016

A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells

por Christensen, Chloe L., Choy, Francis Y. M.
Publicado 2017

Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS

por Christensen, Chloe L., Ashmead, Rhea E., Choy, Francis Y. M.
Publicado 2019

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