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103 results

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Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Powers S, Likhite S, Gadalla KK, Miranda CJ, Huffenberger AJ, Dennys C, Foust KD, Morales P, Pierson CR, Rinaldi F, Perry S, Bolon B, Wein N, Cobb S, Kaspar BK, Meyer KC. Powers S, et al. Among authors: kaspar bk. Mol Ther. 2023 Sep 6;31(9):2767-2782. doi: 10.1016/j.ymthe.2023.07.013. Epub 2023 Jul 22. Mol Ther. 2023. PMID: 37481701 Free PMC article.
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: kaspar bk. Front Genet. 2023 Mar 24;14:1118649. doi: 10.3389/fgene.2023.1118649. eCollection 2023. Front Genet. 2023. PMID: 37035740 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. Among authors: kaspar bk. 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.
Sox11 is an Activity-Regulated Gene with Dentate-Gyrus-Specific Expression Upon General Neural Activation.
von Wittgenstein J, Zheng F, Wittmann MT, Balta EA, Ferrazzi F, Schäffner I, Häberle BM, Valero-Aracama MJ, Koehl M, Miranda CJ, Kaspar BK, Ekici AB, Reis A, Abrous DN, Alzheimer C, Lie DC. von Wittgenstein J, et al. Among authors: kaspar bk. Cereb Cortex. 2020 May 18;30(6):3731-3743. doi: 10.1093/cercor/bhz338. Cereb Cortex. 2020. PMID: 32080705
Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS.
Bravo-Hernandez M, Tadokoro T, Navarro MR, Platoshyn O, Kobayashi Y, Marsala S, Miyanohara A, Juhas S, Juhasova J, Skalnikova H, Tomori Z, Vanicky I, Studenovska H, Proks V, Chen P, Govea-Perez N, Ditsworth D, Ciacci JD, Gao S, Zhu W, Ahrens ET, Driscoll SP, Glenn TD, McAlonis-Downes M, Da Cruz S, Pfaff SL, Kaspar BK, Cleveland DW, Marsala M. Bravo-Hernandez M, et al. Among authors: kaspar bk. Nat Med. 2020 Jan;26(1):118-130. doi: 10.1038/s41591-019-0674-1. Epub 2019 Dec 23. Nat Med. 2020. PMID: 31873312 Free PMC article.
103 results