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AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates.
Mol Ther Methods Clin Dev. 2022 Oct 7;27:281-292. doi: 10.1016/j.omtm.2022.10.004. eCollection 2022 Dec 8.
Mol Ther Methods Clin Dev. 2022.
PMID: 36320411
Free PMC article.
Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis.
Taghian T, Batista AR, Kamper S, Caldwell M, Lilley L, Li H, Rodriguez P, Mesa K, Zheng S, King RM, Gounis MJ, Todeasa S, Maguire A, Martin DR, Sena-Esteves M, Meade TJ, Gray-Edwards HL.
Taghian T, et al. Among authors: todeasa s.
Mol Ther Methods Clin Dev. 2021 Aug 26;23:128-134. doi: 10.1016/j.omtm.2021.08.003. eCollection 2021 Dec 10.
Mol Ther Methods Clin Dev. 2021.
PMID: 34703836
Free PMC article.
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Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
Keeler AM, Zieger M, Todeasa SH, McCall AL, Gifford JC, Birsak S, Choudhury SR, Byrne BJ, Sena-Esteves M, ElMallah MK.
Keeler AM, et al. Among authors: todeasa sh.
Hum Gene Ther. 2019 Jan;30(1):57-68. doi: 10.1089/hum.2018.016. Epub 2018 Jul 25.
Hum Gene Ther. 2019.
PMID: 29901418
Free PMC article.
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Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model.
Stoica L, Todeasa SH, Cabrera GT, Salameh JS, ElMallah MK, Mueller C, Brown RH Jr, Sena-Esteves M.
Stoica L, et al. Among authors: todeasa sh.
Ann Neurol. 2016 Apr;79(4):687-700. doi: 10.1002/ana.24618. Epub 2016 Mar 11.
Ann Neurol. 2016.
PMID: 26891182
Free PMC article.
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