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Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
Cain JT, Likhite S, White KA, Timm DJ, Davis SS, Johnson TB, Dennys-Rivers CN, Rinaldi F, Motti D, Corcoran S, Morales P, Pierson C, Hughes SM, Lee SY, Kaspar BK, Meyer K, Weimer JM. Cain JT, et al. Among authors: timm dj. Mol Ther. 2019 Oct 2;27(10):1836-1847. doi: 10.1016/j.ymthe.2019.06.015. Epub 2019 Jul 10. Mol Ther. 2019. PMID: 31331814 Free PMC article.
A multimodal approach to identify clinically relevant biomarkers to comprehensively monitor disease progression in a mouse model of pediatric neurodegenerative disease.
Johnson TB, Brudvig JJ, Lehtimäki KK, Cain JT, White KA, Bragge T, Rytkönen J, Huhtala T, Timm D, Vihma M, Puoliväli JT, Poutiainen P, Nurmi A, Weimer JM. Johnson TB, et al. Prog Neurobiol. 2020 Jun;189:101789. doi: 10.1016/j.pneurobio.2020.101789. Epub 2020 Mar 18. Prog Neurobiol. 2020. PMID: 32198061
Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease.
Johnson TB, Brudvig JJ, Likhite S, Pratt MA, White KA, Cain JT, Booth CD, Timm DJ, Davis SS, Meyerink B, Pineda R, Dennys-Rivers C, Kaspar BK, Meyer K, Weimer JM. Johnson TB, et al. Among authors: timm dj. Front Genet. 2023 Mar 24;14:1118649. doi: 10.3389/fgene.2023.1118649. eCollection 2023. Front Genet. 2023. PMID: 37035740 Free PMC article.
11 results