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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: hana s. 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.
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.
Chen YA, Kankel MW, Hana S, Lau SK, Zavodszky MI, McKissick O, Mastrangelo N, Dion J, Wang B, Ferretti D, Koske D, Lehman S, Koszka K, McLaughlin H, Liu M, Marshall E, Fabian AJ, Cullen P, Marsh G, Hamann S, Craft M, Sebalusky J, Arnold HM, Driscoll R, Sheehy A, Luo Y, Manca S, Carlile T, Sun C, Sigrist K, McCampbell A, Henderson CE, Lo SC. Chen YA, et al. Among authors: hana s. Gene Ther. 2023 May;30(5):443-454. doi: 10.1038/s41434-022-00375-w. Epub 2022 Dec 1. Gene Ther. 2023. PMID: 36450833 Free PMC article.
Clinical and Molecular Update on the Fourth Reported Family with Hamamy Syndrome.
Mégarbané A, Hana S, Mégarbané H, Castro C, Baulande S, Criqui A, Roëckel-Trevisiol N, Dagher C, Al-Ali MT, Desvignes JP, Mahfoud D, El-Hayek S, Delague V. Mégarbané A, et al. Among authors: hana s. Mol Syndromol. 2021 Oct;12(6):342-350. doi: 10.1159/000517253. Epub 2021 Aug 31. Mol Syndromol. 2021. PMID: 34899143 Free PMC article.
28 results