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Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector.
Curr Gene Ther. 2022;22(3):262-276. doi: 10.2174/1566523221666210916153051.
Curr Gene Ther. 2022.
PMID: 34530708
Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model.
Woodley E, Osmon KJL, Thompson P, Richmond C, Chen Z, Gray SJ, Walia JS.
Woodley E, et al.
Mol Ther Methods Clin Dev. 2018 Oct 26;12:47-57. doi: 10.1016/j.omtm.2018.10.011. eCollection 2019 Mar 15.
Mol Ther Methods Clin Dev. 2018.
PMID: 30534578
Free PMC article.
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Battery of Behavioral Tests Assessing General Locomotion, Muscular Strength, and Coordination in Mice.
Osmon KJ, Vyas M, Woodley E, Thompson P, Walia JS.
Osmon KJ, et al. Among authors: woodley e.
J Vis Exp. 2018 Jan 23;(131):55491. doi: 10.3791/55491.
J Vis Exp. 2018.
PMID: 29443024
Free PMC article.
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Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay-Sachs Disease and Sandhoff Disease Mouse Models.
Kot S, Karumuthil-Melethil S, Woodley E, Zaric V, Thompson P, Chen Z, Lykken E, Keimel JG, Kaemmerer WF, Gray SJ, Walia JS.
Kot S, et al. Among authors: woodley e.
Int J Mol Sci. 2021 Jun 23;22(13):6751. doi: 10.3390/ijms22136751.
Int J Mol Sci. 2021.
PMID: 34201771
Free PMC article.
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Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice.
Osmon KJ, Woodley E, Thompson P, Ong K, Karumuthil-Melethil S, Keimel JG, Mark BL, Mahuran D, Gray SJ, Walia JS.
Osmon KJ, et al. Among authors: woodley e.
Hum Gene Ther. 2016 Jul;27(7):497-508. doi: 10.1089/hum.2016.015.
Hum Gene Ther. 2016.
PMID: 27199088
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