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Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I.
Leier A, Moore M, Liu H, Daniel M, Hyde AM, Messiaen L, Korf BR, Selvakumaran J, Ciszewski L, Lambert L, Foote J, Wallace MR, Kesterson RA, Dickson G, Popplewell L, Wallis D. Leier A, et al. Among authors: korf br. Mol Ther Nucleic Acids. 2022 Mar 15;28:261-278. doi: 10.1016/j.omtn.2022.03.011. eCollection 2022 Jun 14. Mol Ther Nucleic Acids. 2022. PMID: 35433111 Free PMC article.
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: korf br. 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.
Genotype-Phenotype Correlation in NF1: Evidence for a More Severe Phenotype Associated with Missense Mutations Affecting NF1 Codons 844-848.
Koczkowska M, Chen Y, Callens T, Gomes A, Sharp A, Johnson S, Hsiao MC, Chen Z, Balasubramanian M, Barnett CP, Becker TA, Ben-Shachar S, Bertola DR, Blakeley JO, Burkitt-Wright EMM, Callaway A, Crenshaw M, Cunha KS, Cunningham M, D'Agostino MD, Dahan K, De Luca A, Destrée A, Dhamija R, Eoli M, Evans DGR, Galvin-Parton P, George-Abraham JK, Gripp KW, Guevara-Campos J, Hanchard NA, Hernández-Chico C, Immken L, Janssens S, Jones KJ, Keena BA, Kochhar A, Liebelt J, Martir-Negron A, Mahoney MJ, Maystadt I, McDougall C, McEntagart M, Mendelsohn N, Miller DT, Mortier G, Morton J, Pappas J, Plotkin SR, Pond D, Rosenbaum K, Rubin K, Russell L, Rutledge LS, Saletti V, Schonberg R, Schreiber A, Seidel M, Siqveland E, Stockton DW, Trevisson E, Ullrich NJ, Upadhyaya M, van Minkelen R, Verhelst H, Wallace MR, Yap YS, Zackai E, Zonana J, Zurcher V, Claes K, Martin Y, Korf BR, Legius E, Messiaen LM. Koczkowska M, et al. Among authors: korf br. Am J Hum Genet. 2018 Jan 4;102(1):69-87. doi: 10.1016/j.ajhg.2017.12.001. Epub 2017 Dec 28. Am J Hum Genet. 2018. PMID: 29290338 Free PMC article.
286 results