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2010 1
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Page 1
Optimization of Internally Deleted Dystrophin Constructs.
Reza M, Laval SH, Roos A, Carr S, Lochmüller H. Reza M, et al. Hum Gene Ther Methods. 2016 Oct;27(5):174-186. doi: 10.1089/hgtb.2016.026. Epub 2016 Jul 31. Hum Gene Ther Methods. 2016. PMID: 27477497
Lentiviral vectors can be used for full-length dystrophin gene therapy.
Counsell JR, Asgarian Z, Meng J, Ferrer V, Vink CA, Howe SJ, Waddington SN, Thrasher AJ, Muntoni F, Morgan JE, Danos O. Counsell JR, et al. Sci Rep. 2017 Mar 6;7(1):79. doi: 10.1038/s41598-017-00152-5. Sci Rep. 2017. PMID: 28250438 Free PMC article.
A common CHRNE mutation in Brazilian patients with congenital myasthenic syndrome.
Estephan EP, Sobreira CFDR, Dos Santos ACJ, Tomaselli PJ, Marques W Jr, Ortega RPM, Costa MCM, da Silva AMS, Mendonça RH, Caldas VM, Zambon AA, Abath Neto O, Marchiori PE, Heise CO, Reed UC, Azuma Y, Töpf A, Lochmüller H, Zanoteli E. Estephan EP, et al. J Neurol. 2018 Mar;265(3):708-713. doi: 10.1007/s00415-018-8736-8. Epub 2018 Jan 30. J Neurol. 2018. PMID: 29383513 Free PMC article.
19 results