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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2009 1
2010 3
2011 2
2012 1
2013 3
2014 2
2015 7
2017 1
2018 1
2020 2
2021 6
2022 5
2023 4
2024 0

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Page 1
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy.
Waldrop MA, Moore SA, Mathews KD, Darbro BW, Medne L, Finkel R, Connolly AM, Crawford TO, Drachman D, Wein N, Habib AA, Krzesniak-Swinarska MA, Zaidman CM, Collins JJ, Jokela M, Udd B, Day JW, Ortiz-Guerrero G, Statland J, Butterfield RJ, Dunn DM, Weiss RB, Flanigan KM. Waldrop MA, et al. Among authors: wein n. Hum Mutat. 2022 Apr;43(4):511-528. doi: 10.1002/humu.24343. Epub 2022 Mar 7. Hum Mutat. 2022. PMID: 35165973 Free PMC article.
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Powers S, Likhite S, Gadalla KK, Miranda CJ, Huffenberger AJ, Dennys C, Foust KD, Morales P, Pierson CR, Rinaldi F, Perry S, Bolon B, Wein N, Cobb S, Kaspar BK, Meyer KC. Powers S, et al. Among authors: wein n. Mol Ther. 2023 Sep 6;31(9):2767-2782. doi: 10.1016/j.ymthe.2023.07.013. Epub 2023 Jul 22. Mol Ther. 2023. PMID: 37481701 Free article.
U7 snRNA, a Small RNA with a Big Impact in Gene Therapy.
Lesman D, Rodriguez Y, Rajakumar D, Wein N. Lesman D, et al. Among authors: wein n. Hum Gene Ther. 2021 Nov;32(21-22):1317-1329. doi: 10.1089/hum.2021.047. Epub 2021 Aug 26. Hum Gene Ther. 2021. PMID: 34139889 Review.
35 results