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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: mencia a. 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.
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: mencia a. Exp Dermatol. 2013 Sep;22(9):601-3. doi: 10.1111/exd.12203. Exp Dermatol. 2013. PMID: 23947675
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: mencia a. 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: mencia a. 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.
Fibroblast activation and abnormal extracellular matrix remodelling as common hallmarks in three cancer-prone genodermatoses.
Chacón-Solano E, León C, Díaz F, García-García F, García M, Escámez MJ, Guerrero-Aspizua S, Conti CJ, Mencía Á, Martínez-Santamaría L, Llames S, Pévida M, Carbonell-Caballero J, Puig-Butillé JA, Maseda R, Puig S, de Lucas R, Baselga E, Larcher F, Dopazo J, Del Río M. Chacón-Solano E, et al. Among authors: mencia a. Br J Dermatol. 2019 Sep;181(3):512-522. doi: 10.1111/bjd.17698. Epub 2019 Apr 15. Br J Dermatol. 2019. PMID: 30693469 Free PMC article.
Efficient CRISPR-Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome.
Gálvez V, Chacón-Solano E, Bonafont J, Mencía Á, Di WL, Murillas R, Llames S, Vicente A, Del Rio M, Carretero M, Larcher F. Gálvez V, et al. Among authors: mencia a. Mol Ther Methods Clin Dev. 2020 Jun 11;18:280-290. doi: 10.1016/j.omtm.2020.05.031. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32637457 Free PMC article.
41 results