Rha A - Search Results

1

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. Among authors: rha a. Sci Rep. 2022 Dec 14;12(1):21576. doi: 10.1038/s41598-022-25914-8. Sci Rep. 2022. PMID: 36517654 Free PMC article.

2

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: rha a. 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.

3

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: rha ak. 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.

4

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. 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.

5

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. 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.

6

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: rha ak. 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.

7

Polyanion order controls liquid-to-solid phase transition in peptide/nucleic acid co-assembly.

Gordon-Kim C, Rha A, Poppitz GA, Smith-Carpenter J, Luu R, Roberson AB, Conklin R, Blake A, Lynn DG. Gordon-Kim C, et al. Among authors: rha a. Front Mol Biosci. 2022 Nov 14;9:991728. doi: 10.3389/fmolb.2022.991728. eCollection 2022. Front Mol Biosci. 2022. PMID: 36452451 Free PMC article.

8

GM1 Gangliosidosis: Mechanisms and Management.

Rha AK, Maguire AS, Martin DR. Rha AK, et al. Appl Clin Genet. 2021 Apr 9;14:209-233. doi: 10.2147/TACG.S206076. eCollection 2021. Appl Clin Genet. 2021. PMID: 33859490 Free PMC article. Review.

9

Electrostatic Complementarity Drives Amyloid/Nucleic Acid Co-Assembly.

Rha AK, Das D, Taran O, Ke Y, Mehta AK, Lynn DG. Rha AK, et al. Angew Chem Int Ed Engl. 2020 Jan 2;59(1):358-363. doi: 10.1002/anie.201907661. Epub 2019 Nov 14. Angew Chem Int Ed Engl. 2020. PMID: 31617300 Free PMC article.

10

Expanding the informational chemistries of life: peptide/RNA networks.

Taran O, Chen C, Omosun TO, Hsieh MC, Rha A, Goodwin JT, Mehta AK, Grover MA, Lynn DG. Taran O, et al. Among authors: rha a. Philos Trans A Math Phys Eng Sci. 2017 Dec 28;375(2109):20160356. doi: 10.1098/rsta.2016.0356. Philos Trans A Math Phys Eng Sci. 2017. PMID: 29133453

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