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Page 1
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.
A human model of Batten disease shows role of CLN3 in phagocytosis at the photoreceptor-RPE interface.
Tang C, Han J, Dalvi S, Manian K, Winschel L, Volland S, Soto CA, Galloway CA, Spencer W, Roll M, Milliner C, Bonilha VL, Johnson TB, Latchney L, Weimer JM, Augustine EF, Mink JW, Gullapalli VK, Chung M, Williams DS, Singh R. Tang C, et al. Commun Biol. 2021 Feb 5;4(1):161. doi: 10.1038/s42003-021-01682-5. Commun Biol. 2021. PMID: 33547385 Free PMC article.
A CLN6-CLN8 complex recruits lysosomal enzymes at the ER for Golgi transfer.
Bajaj L, Sharma J, di Ronza A, Zhang P, Eblimit A, Pal R, Roman D, Collette JR, Booth C, Chang KT, Sifers RN, Jung SY, Weimer JM, Chen R, Schekman RW, Sardiello M. Bajaj L, et al. J Clin Invest. 2020 Aug 3;130(8):4118-4132. doi: 10.1172/JCI130955. J Clin Invest. 2020. PMID: 32597833 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
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. 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.
32 results