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Combination AAV therapy with galectin-1 and SOD1 downregulation demonstrates superior therapeutic effect in a severe ALS mouse model.
Baird MC, Likhite SB, Vetter TA, Caporale JR, Girard HB, Roussel FS, Howard AE, Schwartz MK, Reed AR, Kaleem A, Zhang X, Meyer KC. Baird MC, et al. Among authors: meyer kc. Mol Ther Methods Clin Dev. 2024 Aug 6;32(3):101312. doi: 10.1016/j.omtm.2024.101312. eCollection 2024 Sep 12. Mol Ther Methods Clin Dev. 2024. PMID: 39257530 Free PMC article.
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans.
Schwartz MK, Likhite S, Vetter TA, Baird MC, McGovern V, Sierra Delgado A, Mendel T, Burghes A, Meyer KC. Schwartz MK, et al. Among authors: meyer kc. Mol Ther Methods Clin Dev. 2023 May 16;30:16-29. doi: 10.1016/j.omtm.2023.05.016. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37746244 Free PMC article.
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.
White KA, Nelvagal HR, Poole TA, Lu B, Johnson TB, Davis S, Pratt MA, Brudvig J, Assis AB, Likhite S, Meyer K, Kaspar BK, Cooper JD, Wang S, Weimer JM. White KA, et al. Mol Ther Methods Clin Dev. 2021 Jan 5;20:497-507. doi: 10.1016/j.omtm.2020.12.014. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2021. PMID: 33665223 Free PMC article.
Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy.
Sinha Ray S, Dutta D, Dennys C, Powers S, Roussel F, Lisowski P, Glažar P, Zhang X, Biswas P, Caporale JR, Rajewsky N, Bickle M, Wein N, Bellen HJ, Likhite S, Marcogliese PC, Meyer KC. Sinha Ray S, et al. Among authors: meyer kc. Cell Rep. 2022 Dec 6;41(10):111751. doi: 10.1016/j.celrep.2022.111751. Cell Rep. 2022. PMID: 36476864 Free article.
AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models.
Murakami Y, Umeshita S, Imanishi K, Yoshioka Y, Ninomiya A, Sunabori T, Likhite S, Koike M, Meyer KC, Kinoshita T. Murakami Y, et al. Among authors: meyer kc. Mol Ther Methods Clin Dev. 2023 Dec 14;32(1):101176. doi: 10.1016/j.omtm.2023.101176. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2023. PMID: 38225934 Free PMC article.
Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS.
Iannitti T, Scarrott JM, Likhite S, Coldicott IRP, Lewis KE, Heath PR, Higginbottom A, Myszczynska MA, Milo M, Hautbergue GM, Meyer K, Kaspar BK, Ferraiuolo L, Shaw PJ, Azzouz M. Iannitti T, et al. Mol Ther Nucleic Acids. 2018 Sep 7;12:75-88. doi: 10.1016/j.omtn.2018.04.015. Epub 2018 May 3. Mol Ther Nucleic Acids. 2018. PMID: 30195799 Free PMC article.
231 results