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Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.
Cavazza A, Hendel A, Bak RO, Rio P, Güell M, Lainšček D, Arechavala-Gomeza V, Peng L, Hapil FZ, Harvey J, Ortega FG, Gonzalez-Martinez C, Lederer CW, Mikkelsen K, Gasiunas G, Kalter N, Gonçalves MAFV, Petersen J, Garanto A, Montoliu L, Maresca M, Seemann SE, Gorodkin J, Mazini L, Sanchez R, Rodriguez-Madoz JR, Maldonado-Pérez N, Laura T, Schmueck-Henneresse M, Maccalli C, Grünewald J, Carmona G, Kachamakova-Trojanowska N, Miccio A, Martin F, Turchiano G, Cathomen T, Luo Y, Tsai SQ, Benabdellah K; COST Action CA21113. Cavazza A, et al. Among authors: goncalves mafv. Mol Ther Nucleic Acids. 2023 Oct 29;34:102066. doi: 10.1016/j.omtn.2023.102066. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 38034032 Free PMC article. Review.
Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes.
Latella MC, Cocchiarella F, De Rosa L, Turchiano G, Gonçalves MAFV, Larcher F, De Luca M, Recchia A. Latella MC, et al. Among authors: goncalves mafv. J Invest Dermatol. 2017 Apr;137(4):836-844. doi: 10.1016/j.jid.2016.11.038. Epub 2016 Dec 24. J Invest Dermatol. 2017. PMID: 28027893 Free article.
AAV-vectored base editor trans-splicing delivers dystrophin repair.
Li Z, Gonçalves MAFV. Li Z, et al. Among authors: goncalves mafv. Mol Ther Nucleic Acids. 2023 May 30;32:900-902. doi: 10.1016/j.omtn.2023.05.013. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37346982 Free PMC article. No abstract available.
63 results