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American Journal of Medical Genetics|October 3, 2002
FGFs, their receptors, and human limb malformations: clinical and molecular correlationsAndrew O M Wilkie, Susannah J Patey, Shih-Hsin Kan, et al.
Molecular Therapy. Methods & Clinical Development|August 14, 2018
Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B MiceDon Clarke, Yewande Pearse, Shih-Hsin Kan, et al.
American Journal of Human Genetics|March 22, 2003
Missense mutations in the homeodomain of HOXD13 are associated with brachydactyly types D and EDavid Johnson, Shih-Hsin Kan, Michael Oldridge, et al.
Pediatric Research|September 5, 2013
Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patientsMoin Vera, Steven Le, Shih-Hsin Kan, et al.
Molecular Therapy. Methods & Clinical Development|January 7, 2025
Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I miceCaitlin C Calhoun, Shih-Hsin Kan, Alexander E Stover, et al.
Scientific Reports|June 27, 2020
CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weaknessJeffrey Y Huang, Shih-Hsin Kan, Emilie K Sandfeld, et al.
Lipids|June 16, 2020
Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I MiceSteven Q Le, Igor Nestrasil, Shih-Hsin Kan, et al.
Scientific Reports|December 14, 2022
CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe diseaseShih-Hsin Kan, Jeffrey Y Huang, Jerry Harb, 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.
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.
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