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An RNA-targeted therapy for dystrophic epidermolysis bullosa.
Peking P, Koller U, Duarte B, Murillas R, Wolf S, Maetzig T, Rothe M, Kocher T, García M, Brachtl G, Schambach A, Larcher F, Reichelt J, Bauer JW, Murauer EM. Peking P, et al. Among authors: murillas r. Nucleic Acids Res. 2017 Sep 29;45(17):10259-10269. doi: 10.1093/nar/gkx669. Nucleic Acids Res. 2017. PMID: 28973459 Free PMC article.
Keratinocyte cell lines derived from severe generalized recessive epidermolysis bullosa patients carrying a highly recurrent COL7A1 homozygous mutation: models to assess cell and gene therapies in vitro and in vivo.
Chamorro C, Almarza D, Duarte B, Llames SG, Murillas R, García M, Cigudosa JC, Espinosa-Hevia L, Escámez MJ, Mencía A, Meana A, García-Escudero R, Moro R, Conti CJ, Del Río M, Larcher F. Chamorro C, et al. Among authors: murillas r. Exp Dermatol. 2013 Sep;22(9):601-3. doi: 10.1111/exd.12203. Exp Dermatol. 2013. PMID: 23947675
Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.
Bonafont J, Mencía A, Chacón-Solano E, Srifa W, Vaidyanathan S, Romano R, Garcia M, Hervás-Salcedo R, Ugalde L, Duarte B, Porteus MH, Del Rio M, Larcher F, Murillas R. Bonafont J, et al. Among authors: murillas r. Mol Ther. 2021 Jun 2;29(6):2008-2018. doi: 10.1016/j.ymthe.2021.02.019. Epub 2021 Feb 18. Mol Ther. 2021. PMID: 33609734 Free PMC article.
Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem Cells.
Mencía Á, Chamorro C, Bonafont J, Duarte B, Holguin A, Illera N, Llames SG, Escámez MJ, Hausser I, Del Río M, Larcher F, Murillas R. Mencía Á, et al. Among authors: murillas r. Mol Ther Nucleic Acids. 2018 Jun 1;11:68-78. doi: 10.1016/j.omtn.2018.01.009. Epub 2018 Jan 31. Mol Ther Nucleic Acids. 2018. PMID: 29858091 Free PMC article.
Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing.
Bonafont J, Mencía Á, García M, Torres R, Rodríguez S, Carretero M, Chacón-Solano E, Modamio-Høybjør S, Marinas L, León C, Escamez MJ, Hausser I, Del Río M, Murillas R, Larcher F. Bonafont J, et al. Among authors: murillas r. Mol Ther. 2019 May 8;27(5):986-998. doi: 10.1016/j.ymthe.2019.03.007. Epub 2019 Mar 15. Mol Ther. 2019. PMID: 30930113 Free PMC article.
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors.
García M, Bonafont J, Martínez-Palacios J, Xu R, Turchiano G, Svensson S, Thrasher AJ, Larcher F, Del Rio M, Hernández-Alcoceba R, Garín MI, Mencía Á, Murillas R. García M, et al. Among authors: murillas r. Mol Ther Methods Clin Dev. 2022 Sep 16;27:96-108. doi: 10.1016/j.omtm.2022.09.005. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36212909 Free PMC article.
28 results