Wein N - Search Results

1

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: wein n. 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.

2

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: wein n. 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.

3

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping.

Simmons TR, Vetter TA, Huang N, Vulin-Chaffiol A, Wein N, Flanigan KM. Simmons TR, et al. Among authors: wein n. Mol Ther Methods Clin Dev. 2021 Mar 23;21:325-340. doi: 10.1016/j.omtm.2021.03.014. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33898631 Free PMC article.

4

Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications.

Wein N, Dunn DM, Waldrop MA, Gushchina LV, Frair EC, Weiss RB, Flanigan KM. Wein N, et al. Hum Gene Ther. 2021 Nov;32(21-22):1346-1359. doi: 10.1089/hum.2020.315. Epub 2021 Jul 20. Hum Gene Ther. 2021. PMID: 34060935

5

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. 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.

6

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: wein n. 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.

7

Genetics and emerging treatments for Duchenne and Becker muscular dystrophy.

Wein N, Alfano L, Flanigan KM. Wein N, et al. Pediatr Clin North Am. 2015 Jun;62(3):723-42. doi: 10.1016/j.pcl.2015.03.008. Epub 2015 Apr 20. Pediatr Clin North Am. 2015. PMID: 26022172 Review.

8

The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development.

Vulin A, Wein N, Simmons TR, Rutherford AM, Findlay AR, Yurkoski JA, Kaminoh Y, Flanigan KM. Vulin A, et al. Among authors: wein n. Neuromuscul Disord. 2015 Nov;25(11):827-34. doi: 10.1016/j.nmd.2015.08.005. Epub 2015 Aug 11. Neuromuscul Disord. 2015. PMID: 26365037

9

Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach.

Wein N, Vulin A, Findlay AR, Gumienny F, Huang N, Wilton SD, Flanigan KM. Wein N, et al. J Neuromuscul Dis. 2017;4(3):199-207. doi: 10.3233/JND-170233. J Neuromuscul Dis. 2017. PMID: 28869484

10

Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.

Findlay AR, Wein N, Kaminoh Y, Taylor LE, Dunn DM, Mendell JR, King WM, Pestronk A, Florence JM, Mathews KD, Finkel RS, Swoboda KJ, Howard MT, Day JW, McDonald C, Nicolas A, Le Rumeur E, Weiss RB, Flanigan KM; United Dystrophinopathy Project. Findlay AR, et al. Among authors: wein n. Ann Neurol. 2015 Apr;77(4):668-74. doi: 10.1002/ana.24365. Epub 2015 Mar 2. Ann Neurol. 2015. PMID: 25612243 Free PMC article.

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