Shih-hsin Kan

Title(s)Assistant Adjunct Professor, Pediatrics
SchoolSchool of Medicine
Address1001 Health Sciences Road
Irvine CA 92697-3950
Phone(714) 509-3337
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    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Neonatal gene therapy with AAV2/8-LSPhGAA improves hypertrophic cardiomyopathy in the Gaac.1826dupA knock-in murine model. Mol Genet Metab. 2026 Jul 04; 149(1-2):110204. Kan SH, Harb JF, Li S, Kliman A, Andrade-Heckman P, Christensen CL, Han SO, Koeberl DD, Wang RY. PMID: 42442016.
      View in: PubMed   Mentions:    Fields:    
    2. First-in-human intracisternal dosing of RGX-111 in severe MPS I is well tolerated and generates sustained neurodevelopment without HSCT. Mol Ther. 2026 Jun 03; 34(6):3217-3223. Wang RY, Movsesyan N, Kan SH, Beydoun T, Taylor M, Chang RC, Phillips D, Burke J, Gilmor M, Cho Y, Falabella P, Pisani L. PMID: 41966056.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    3. Longitudinal characterization of Gaac.1826dupA mice reveals the cardiac, myopathic and biochemical phenotypes of Pompe disease. Dis Model Mech. 2026 Mar 01; 19(3). Harb JF, Kan SH, Christensen CL, Rha AK, Andrade-Heckman P, Kliman A, Padilla A, Holbrook C, Huang JY, Koeberl DD, Wang RY. PMID: 41622854; PMCID: PMC13035063.
      View in: PubMed   Mentions:    Fields:    Translation:AnimalsCells
    4. Antibodies to recombinant human alpha-L-iduronidase prevent disease correction in cortical bone in MPS I mice. Mol Ther Methods Clin Dev. 2025 Mar 13; 33(1):101405. Hurt SC, Le SQ, Kan SH, Bui QD, Brodt MD, Dickson PI. PMID: 40123743; PMCID: PMC11928967.
      View in: PubMed   Mentions:
    5. Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice. Mol Ther Methods Clin Dev. 2024 Dec 12; 32(4):101367. Calhoun CC, Kan SH, Stover AE, Harb JF, Monuki ES, Wang RY, Schwartz PH. PMID: 39764351; PMCID: PMC11701249.
      View in: PubMed   Mentions: 1  
    6. Base editing of the GLB1 gene is therapeutic in GM1 gangliosidosis patient-derived cells. Mol Genet Metab. 2024 Sep-Oct; 143(1-2):108568. Rha AK, Kan SH, Andrade-Heckman P, Christensen CL, Harb JF, Wang RY. PMID: 39303319.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    7. Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testing. Stem Cell Res. 2024 12; 81:103552. Rha AK, Christensen CL, Kan SH, Harb JF, Andrade-Heckman P, Wang RY. PMID: 39303321.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    8. Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells. Mol Ther Nucleic Acids. 2024 Jun 11; 35(2):102220. Christensen CL, Kan SH, Andrade-Heckman P, Rha AK, Harb JF, Wang RY. PMID: 38948331; PMCID: PMC11214518.
      View in: PubMed   Mentions: 2  
    9. Intra-Articular AAV9 α-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I. Adv Cell Gene Ther. 2023 Jan; 2023(1). Wang RY, Kan SH, Zhang H, Smith JD, Aminian A, Snella E, Jens JK, Young SP, Dickson PI, Matthew Ellinwood N. PMID: 40881820; PMCID: PMC12382353.
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    10. Base editing corrects the common Salla disease SLC17A5 c.115C>T variant. Mol Ther Nucleic Acids. 2023 Dec 12; 34:102022. Harb JF, Christensen CL, Kan SH, Rha AK, Andrade-Heckman P, Pollard L, Steet R, Huang JY, Wang RY. PMID: 37727271; PMCID: PMC10506058.
      View in: PubMed   Mentions: 5  
    11. Generation of two induced pluripotent stem cell lines (CHOCi002-A and CHOCi003-A) from Pompe disease patients with compound heterozygous mutations in the GAA gene. Stem Cell Res. 2023 06; 69:103117. Christensen C, Heckman P, Rha A, Kan SH, Harb J, Wang R. PMID: 37167752; PMCID: PMC10281086.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    12. 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 disease. Sci Rep. 2022 12 14; 12(1):21576. Kan SH, Huang JY, Harb J, Rha A, Dalton ND, Christensen C, Chan Y, Davis-Turak J, Neumann J, Wang RY. PMID: 36517654; PMCID: PMC9751086.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansAnimals
    13. Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease. Mol Ther Methods Clin Dev. 2022 Dec 08; 27:452-463. Pearse Y, Clarke D, Kan SH, Le SQ, Sanghez V, Luzzi A, Pham I, Nih LR, Cooper JD, Dickson PI, Iacovino M. PMID: 36419468; PMCID: PMC9672419.
      View in: PubMed   Mentions: 4  
    14. Impaired mitophagy in Sanfilippo a mice causes hypertriglyceridemia and brown adipose tissue activation. J Biol Chem. 2022 08; 298(8):102159. Tillo M, Lamanna WC, Dwyer CA, Sandoval DR, Pessentheiner AR, Al-Azzam N, Sarrazin S, Gonzales JC, Kan SH, Andreyev AY, Schultheis N, Thacker BE, Glass CA, Dickson PI, Wang RY, Selleck SB, Esko JD, Gordts PLSM. PMID: 35750212; PMCID: PMC9364035.
      View in: PubMed   Mentions: 4     Fields:    Translation:Animals
    15. Neuropathology of murine Sanfilippo D syndrome. Mol Genet Metab. 2021 12; 134(4):323-329. Takahashi K, Le SQ, Kan SH, Jansen MJ, Dickson PI, Cooper JD. PMID: 34844863.
      View in: PubMed   Mentions: 5     Fields:    Translation:AnimalsCells
    16. Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome. Mol Genet Metab. 2021 06; 133(2):185-192. Kan SH, Elsharkawi I, Le SQ, Prill H, Mangini L, Cooper JD, Lawrence R, Sands MS, Crawford BE, Dickson PI. PMID: 33839004; PMCID: PMC8195848.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimals
    17. Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice. Mol Pharm. 2021 01 04; 18(1):214-227. Wang F, Moen DR, Sauni C, Kan SH, Li S, Le SQ, Lomenick B, Zhang X, Ekins S, Singamsetty S, Wood J, Dickson PI, Chou TF. PMID: 33320673; PMCID: PMC8362844.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansAnimalsCells
    18. CRISPR-Cas9 generated Pompe knock-in murine model exhibits early-onset hypertrophic cardiomyopathy and skeletal muscle weakness. Sci Rep. 2020 Jun 25; 10(1):10321. Huang JY, Kan SH, Sandfeld EK, Dalton ND, Rangel AD, Chan Y, Davis-Turak J, Neumann J, Wang RY. PMID: 32587263; PMCID: PMC7316971.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansAnimals
    19. Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice. Lipids. 2020 11; 55(6):627-637. Le SQ, Nestrasil I, Kan SH, Egeland M, Cooper JD, Elashoff D, Guo R, Tolar J, Yee JK, Dickson PI. PMID: 32537944; PMCID: PMC7992919.
      View in: PubMed   Mentions: 3     Fields:    Translation:AnimalsCells
    20. Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice. Behav Brain Res. 2016 10 01; 312:265-71. Kan SH, Le SQ, Bui QD, Benedict B, Cushman J, Sands MS, Dickson PI. PMID: 27340089; PMCID: PMC4970944.
      View in: PubMed   Mentions: 7     Fields:    Translation:Animals
    21. Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I. Exp Neurol. 2015 Nov; 273:1-10. Provenzale JM, Nestrasil I, Chen S, Kan SH, Le SQ, Jens JK, Snella EM, Vondrak KN, Yee JK, Vite CH, Elashoff D, Duan L, Wang RY, Ellinwood NM, Guzman MA, Shapiro EG, Dickson PI. PMID: 26222335; PMCID: PMC4644459.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansAnimalsCells
    22. A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I. Mol Ther Methods Clin Dev. 2015; 2:14068. Mendez DC, Stover AE, Rangel AD, Brick DJ, Nethercott HE, Torres MA, Khalid O, Wong AM, Cooper JD, Jester JV, Monuki ES, McGuire C, Le SQ, Kan SH, Dickson PI, Schwartz PH. PMID: 26052536; PMCID: PMC4449030.
      View in: PubMed   Mentions: 11  
    23. Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I. Mol Genet Metab. 2014 Aug; 112(4):286-93. Wang RY, Aminian A, McEntee MF, Kan SH, Simonaro CM, Lamanna WC, Lawrence R, Ellinwood NM, Guerra C, Le SQ, Dickson PI, Esko JD. PMID: 24951454; PMCID: PMC4122635.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansAnimalsCells
    24. Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients. Pediatr Res. 2013 Dec; 74(6):712-20. Vera M, Le S, Kan SH, Garban H, Naylor D, Mlikotic A, Kaitila I, Harmatz P, Chen A, Dickson P. PMID: 24002329; PMCID: PMC3855632.
      View in: PubMed   Mentions: 12     Fields:    Translation:Humans
    25. Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I. Comp Med. 2013 Apr; 63(2):163-73. Vite CH, Nestrasil I, Mlikotic A, Jens JK, Snella EM, Gross W, Shapiro EG, Kovac V, Provenzale JM, Chen S, Le SQ, Kan SH, Banakar S, Wang RY, Haskins ME, Ellinwood NM, Dickson PI. PMID: 23582423; PMCID: PMC3625057.
      View in: PubMed   Mentions: 25     Fields:    Translation:Animals
    26. Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro. Biotechnol Appl Biochem. 2011 Nov-Dec; 58(6):391-6. Tippin BL, Troitskaya L, Kan SH, Todd AK, Le SQ, Dickson PI. PMID: 22172101; PMCID: PMC3293367.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCells
    27. Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I. Mol Genet Metab. 2010 Oct-Nov; 101(2-3):115-22. Dickson PI, Hanson S, McEntee MF, Vite CH, Vogler CA, Mlikotic A, Chen AH, Ponder KP, Haskins ME, Tippin BL, Le SQ, Passage MB, Guerra C, Dierenfeld A, Jens J, Snella E, Kan SH, Ellinwood NM. PMID: 20655780; PMCID: PMC2950221.
      View in: PubMed   Mentions: 27     Fields:    Translation:HumansAnimals
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