Kan SH - Search Results

1

Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testing.

Rha AK, Christensen CL, Kan SH, Harb JF, Andrade-Heckman P, Wang RY. Rha AK, et al. Among authors: kan sh. Stem Cell Res. 2024 Sep 7;81:103552. doi: 10.1016/j.scr.2024.103552. Online ahead of print. Stem Cell Res. 2024. PMID: 39303321 Free article.

2

Base editing of the GLB1 gene is therapeutic in GM1 gangliosidosis patient-derived cells.

Rha AK, Kan SH, Andrade-Heckman P, Christensen CL, Harb JF, Wang RY. Rha AK, et al. Among authors: kan sh. Mol Genet Metab. 2024 Sep-Oct;143(1-2):108568. doi: 10.1016/j.ymgme.2024.108568. Epub 2024 Sep 7. Mol Genet Metab. 2024. PMID: 39303319 Free article.

3

Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells.

Christensen CL, Kan SH, Andrade-Heckman P, Rha AK, Harb JF, Wang RY. Christensen CL, et al. Among authors: kan sh. Mol Ther Nucleic Acids. 2024 May 21;35(2):102220. doi: 10.1016/j.omtn.2024.102220. eCollection 2024 Jun 11. Mol Ther Nucleic Acids. 2024. PMID: 38948331 Free PMC article.

4

Combining angiotensin receptor blockade and enzyme replacement therapy for vascular disease in mucopolysaccharidosis type I.

Hurt SC, Vera MU, Le SQ, Kan SH, Bui Q, Dickson PI. Hurt SC, et al. Among authors: kan sh. Mol Genet Metab Rep. 2023 Dec 14;38:101036. doi: 10.1016/j.ymgmr.2023.101036. eCollection 2024 Mar. Mol Genet Metab Rep. 2023. PMID: 38173710 Free PMC article.

5

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

Harb JF, Christensen CL, Kan SH, Rha AK, Andrade-Heckman P, Pollard L, Steet R, Huang JY, Wang RY. Harb JF, et al. Among authors: kan sh. Mol Ther Nucleic Acids. 2023 Aug 26;34:102022. doi: 10.1016/j.omtn.2023.08.024. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 37727271 Free PMC article.

6

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.

Christensen C, Heckman P, Rha A, Kan SH, Harb J, Wang R. Christensen C, et al. Among authors: kan sh. Stem Cell Res. 2023 Jun;69:103117. doi: 10.1016/j.scr.2023.103117. Epub 2023 May 6. Stem Cell Res. 2023. PMID: 37167752 Free PMC article.

7

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.

Kan SH, Huang JY, Harb J, Rha A, Dalton ND, Christensen C, Chan Y, Davis-Turak J, Neumann J, Wang RY. Kan SH, et al. Sci Rep. 2022 Dec 14;12(1):21576. doi: 10.1038/s41598-022-25914-8. Sci Rep. 2022. PMID: 36517654 Free PMC article.

8

Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease.

Pearse Y, Clarke D, Kan SH, Le SQ, Sanghez V, Luzzi A, Pham I, Nih LR, Cooper JD, Dickson PI, Iacovino M. Pearse Y, et al. Among authors: kan sh. Mol Ther Methods Clin Dev. 2022 Oct 27;27:452-463. doi: 10.1016/j.omtm.2022.10.013. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36419468 Free PMC article.

9

Impaired mitophagy in Sanfilippo a mice causes hypertriglyceridemia and brown adipose tissue activation.

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. Tillo M, et al. Among authors: kan sh. J Biol Chem. 2022 Aug;298(8):102159. doi: 10.1016/j.jbc.2022.102159. Epub 2022 Jun 22. J Biol Chem. 2022. PMID: 35750212 Free PMC article.

10

Neuropathology of murine Sanfilippo D syndrome.

Takahashi K, Le SQ, Kan SH, Jansen MJ, Dickson PI, Cooper JD. Takahashi K, et al. Among authors: kan sh. Mol Genet Metab. 2021 Dec;134(4):323-329. doi: 10.1016/j.ymgme.2021.11.010. Epub 2021 Nov 24. Mol Genet Metab. 2021. PMID: 34844863

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