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Mutation-Directed Therapeutics for Neurofibromatosis Type I.
Leier A, Bedwell DM, Chen AT, Dickson G, Keeling KM, Kesterson RA, Korf BR, Marquez Lago TT, Müller UF, Popplewell L, Zhou J, Wallis D. Leier A, et al. Among authors: popplewell l. Mol Ther Nucleic Acids. 2020 Jun 5;20:739-753. doi: 10.1016/j.omtn.2020.04.012. Epub 2020 Apr 29. Mol Ther Nucleic Acids. 2020. PMID: 32408052 Free PMC article. Review.
Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.
Koo T, Malerba A, Athanasopoulos T, Trollet C, Boldrin L, Ferry A, Popplewell L, Foster H, Foster K, Dickson G. Koo T, et al. Among authors: popplewell l. Hum Gene Ther. 2011 Nov;22(11):1379-88. doi: 10.1089/hum.2011.020. Epub 2011 May 25. Hum Gene Ther. 2011. PMID: 21453126 Free PMC article.
168 results