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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: liu h. 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.
snRNA-seq of human cutaneous neurofibromas before and after selumetinib treatment implicates role of altered Schwann cell states, inter-cellular signaling, and extracellular matrix in treatment response.
Church C, Fay CX, Kriukov E, Liu H, Cannon A, Baldwin LA, Crossman DK, Korf B, Wallace MR, Gross AM, Widemann BC, Kesterson RA, Baranov P, Wallis D. Church C, et al. Among authors: liu h. Acta Neuropathol Commun. 2024 Jun 21;12(1):102. doi: 10.1186/s40478-024-01821-z. Acta Neuropathol Commun. 2024. PMID: 38907342 Free PMC article.
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