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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: hamann 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.
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: hamann 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.
Adeno-Associated Virus-Mediated Dorsal Root Ganglion Toxicity in the New Zealand White Rabbit.
Tien E, Grubor B, Kirkland M, Chan SJ, van der Munnik N, Xu W, Henry K, Hamann S, Wei C, Lee WH, Gianni D, Brennecke A, Nambiar K, Chen J, Liu B, Shen S, Tremblay C, Plowey ED, Trapa P, Fikes J, Suh J, Morris D. Tien E, et al. Among authors: hamann s. Toxicol Pathol. 2024 Jan;52(1):35-54. doi: 10.1177/01926233241229808. Epub 2024 Feb 22. Toxicol Pathol. 2024. PMID: 38385340
The BONSAI (Brain and Optic Nerve Study with Artificial Intelligence) deep learning system can accurately identify pediatric papilledema on standard ocular fundus photographs.
Lin MY, Najjar RP, Tang Z, Cioplean D, Dragomir M, Chia A, Patil A, Vasseneix C, Peragallo JH, Newman NJ, Biousse V, Milea D; BONSAI (Brain and Optic Nerve Study with Artificial Intelligence) group. Lin MY, et al. J AAPOS. 2024 Feb;28(1):103803. doi: 10.1016/j.jaapos.2023.10.005. Epub 2024 Jan 10. J AAPOS. 2024. PMID: 38216117
355 results