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Diseases (Basel, Switzerland)|September 22, 2017
A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem CellsChloe L Christensen, Francis Y M Choy
Diseases (Basel, Switzerland)|June 29, 2019
Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNSChloe L Christensen, Rhea E Ashmead, Francis Y M Choy
Molecular Therapy. Nucleic Acids|July 1, 2024
Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cellsChloe L Christensen, Shih-Hsin Kan, Perla Andrade-Heckman, et al.
Stem Cell Research|September 20, 2024
Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testingAllisandra K Rha, Chloe L Christensen, Shih-Hsin Kan, et al.
Molecular Genetics and Metabolism|September 20, 2024
Base editing of the GLB1 gene is therapeutic in GM1 gangliosidosis patient-derived cellsAllisandra K Rha, Shih-Hsin Kan, Perla Andrade-Heckman, et al.
Molecular Therapy. Nucleic Acids|September 20, 2023
Base editing corrects the common Salla disease SLC17A5 c.115C>T variantJerry F Harb, Chloe L Christensen, Shih-Hsin Kan, et al.
Molecular Genetics and Metabolism|July 13, 2026
Neonatal gene therapy with AAV2/8-LSPhGAA improves hypertrophic cardiomyopathy in the Gaac.1826dupA knock-in murine modelShih-Hsin Kan, Jerry F Harb, Songtao Li, et al.
Disease Models & Mechanisms|February 2, 2026
Longitudinal characterization of Gaac.1826dupA mouse reveals cardiac, myopathic, biochemical phenotypes of Pompe diseaseJerry F Harb, Shih-Hsin Kan, Chloe L Christensen, et al.
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