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Page 1
Human SNORA31 variations impair cortical neuron-intrinsic immunity to HSV-1 and underlie herpes simplex encephalitis.
Lafaille FG, Harschnitz O, Lee YS, Zhang P, Hasek ML, Kerner G, Itan Y, Ewaleifoh O, Rapaport F, Carlile TM, Carter-Timofte ME, Paquet D, Dobbs K, Zimmer B, Gao D, Rojas-Duran MF, Kwart D, Rattina V, Ciancanelli MJ, McAlpine JL, Lorenzo L, Boucherit S, Rozenberg F, Halwani R, Henry B, Amenzoui N, Alsum Z, Marques L, Church JA, Al-Muhsen S, Tardieu M, Bousfiha AA, Paludan SR, Mogensen TH, Quintana-Murci L, Tessier-Lavigne M, Smith GA, Notarangelo LD, Studer L, Gilbert W, Abel L, Casanova JL, Zhang SY. Lafaille FG, et al. Among authors: carlile tm. Nat Med. 2019 Dec;25(12):1873-1884. doi: 10.1038/s41591-019-0672-3. Epub 2019 Dec 5. Nat Med. 2019. PMID: 31806906 Free PMC article.
Dorsal root ganglion toxicity after AAV intra-CSF delivery of a RNAi expression construct into non-human primates and mice.
Hawley ZCE, Pardo ID, Cao S, Zavodszky MI, Casey F, Ferber K, Luo Y, Hana S, Chen SK, Doherty J, Costa R, Cullen P, Liu Y, Carlile TM, Chowdhury T, Doyle B, Clarner P, Mangaudis K, Guilmette E, Bourque S, Koske D, Nadella MVP, Trapa P, Hawes ML, Raitcheva D, Lo SC. Hawley ZCE, et al. Among authors: carlile tm. Mol Ther. 2025 Jan 8;33(1):215-234. doi: 10.1016/j.ymthe.2024.11.029. Epub 2024 Nov 19. Mol Ther. 2025. PMID: 39563026 Free PMC article.
Inhibition of immune response reduces pathology in dorsal root ganglia and peripheral nerves in cynomolgus macaques following AAV gene therapy.
Grubor B, Henry KL, Chan SJ, Sheehan M, Shah A, Pellerin A, Bai J, Nadella P, Bommegowda S, Cullen P, Tien E, Chen V, van der Munnik NP, White-Hunt S, Plowey ED, Hamann S, Guise AJ, Xu S, Kirkland M, Doherty J, Lyashenko E, Bhat G, Glajch K, Lo SC, Gianni D, Clarner P, Gagnon J, Chu J, Nambiar K, Lulla M, Zheng F, Weldeab A, Bartlett D, Gutnick A, Reynolds T, Zhu K, Huh D, Carlile TM, Fikes J, Trapa P, Suh J, Morris D, Burkly L. Grubor B, et al. Among authors: carlile tm. Mol Ther Methods Clin Dev. 2025 Nov 14;33(4):101643. doi: 10.1016/j.omtm.2025.101643. eCollection 2025 Dec 11. Mol Ther Methods Clin Dev. 2025. PMID: 41404412 Free PMC article.
MOG autoantibodies trigger a tightly-controlled FcR and BTK-driven microglia proliferative response.
Pellerin K, Rubino SJ, Burns JC, Smith BA, McCarl CA, Zhu J, Jandreski L, Cullen P, Carlile TM, Li A, Rebollar JV, Sybulski J, Reynolds TL, Zhang B, Basile R, Tang H, Harp CP, Pellerin A, Silbereis J, Franchimont N, Cahir-McFarland E, Ransohoff RM, Cameron TO, Mingueneau M. Pellerin K, et al. Among authors: carlile tm. Brain. 2021 Sep 4;144(8):2361-2374. doi: 10.1093/brain/awab231. Brain. 2021. PMID: 34145876
Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection.
Chen SK, Hawley ZCE, Zavodszky MI, Hana S, Ferretti D, Grubor B, Hawes M, Xu S, Hamann S, Marsh G, Cullen P, Challa R, Carlile TM, Zhang H, Lee WH, Peralta A, Clarner P, Wei C, Koszka K, Gao F, Lo SC. Chen SK, et al. Among authors: carlile tm. Mol Ther Nucleic Acids. 2023 Oct 16;34:102057. doi: 10.1016/j.omtn.2023.102057. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 37928442 Free PMC article.
17 results