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Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies.
Neuropathol Appl Neurobiol. 2022 Apr;48(3):e12785. doi: 10.1111/nan.12785. Epub 2021 Dec 28.
Neuropathol Appl Neurobiol. 2022.
PMID: 34847621
Free PMC article.
Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy.
Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM.
Gushchina LV, et al. Among authors: bradley aj.
Mol Ther Nucleic Acids. 2022 Nov 9;30:479-492. doi: 10.1016/j.omtn.2022.10.025. eCollection 2022 Dec 13.
Mol Ther Nucleic Acids. 2022.
PMID: 36420217
Free PMC article.
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Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping.
Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM.
Gushchina LV, et al. Among authors: bradley aj.
Hum Gene Ther. 2021 Sep;32(17-18):882-894. doi: 10.1089/hum.2020.286. Epub 2021 May 7.
Hum Gene Ther. 2021.
PMID: 33406986
Free PMC article.
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Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.
Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM.
Wein N, et al. Among authors: bradley aj.
Mol Ther Methods Clin Dev. 2022 Jul 11;26:279-293. doi: 10.1016/j.omtm.2022.07.005. eCollection 2022 Sep 8.
Mol Ther Methods Clin Dev. 2022.
PMID: 35949298
Free PMC article.
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Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model.
Gushchina LV, Bradley AJ, Vetter TA, Lay JW, Rohan NL, Frair EC, Wein N, Flanigan KM.
Gushchina LV, et al. Among authors: bradley aj.
Mol Ther Methods Clin Dev. 2023 Oct 26;31:101144. doi: 10.1016/j.omtm.2023.101144. eCollection 2023 Dec 14.
Mol Ther Methods Clin Dev. 2023.
PMID: 38027058
Free PMC article.
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