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Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis.
Int J Mol Sci. 2023 Sep 27;24(19):14611. doi: 10.3390/ijms241914611.
Int J Mol Sci. 2023.
PMID: 37834060
Free PMC article.
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.
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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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.
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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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. Among authors: osmon kjl.
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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Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector.
Osmon KJ, Thompson P, Woodley E, Karumuthil-Melethil S, Heindel C, Keimel JG, Kaemmerer WF, Gray SJ, Walia JS.
Osmon KJ, et al.
Curr Gene Ther. 2022;22(3):262-276. doi: 10.2174/1566523221666210916153051.
Curr Gene Ther. 2022.
PMID: 34530708
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Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study.
Ryckman AE, Deschenes NM, Quinville BM, Osmon KJL, Mitchell M, Chen Z, Gray SJ, Walia JS.
Ryckman AE, et al. Among authors: osmon kjl.
Mol Ther Methods Clin Dev. 2023 Dec 5;32(1):101168. doi: 10.1016/j.omtm.2023.101168. eCollection 2024 Mar 14.
Mol Ther Methods Clin Dev. 2023.
PMID: 38205442
Free PMC article.
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