Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes

por Chamorro, Cristina, Mencía, Angeles, Almarza, David, Duarte, Blanca, Büning, Hildegard, Sallach, Jessica, Hausser, Ingrid, Del Río, Marcela, Larcher, Fernando, Murillas, Rodolfo
Publicado 2016

Efficient CRISPR-Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome

por Gálvez, Victoria, Chacón-Solano, Esteban, Bonafont, Jose, Mencía, Ángeles, Di, Wei-Li, Murillas, Rodolfo, Llames, Sara, Vicente, Asunción, Del Rio, Marcela, Carretero, Marta, Larcher, Fernando
Publicado 2020

Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem Cells

por Mencía, Ángeles, Chamorro, Cristina, Bonafont, Jose, Duarte, Blanca, Holguin, Almudena, Illera, Nuria, Llames, Sara G., Escámez, Maria José, Hausser, Ingrid, Del Río, Marcela, Larcher, Fernando, Murillas, Rodolfo
Publicado 2018

Three deaf mice: mouse models for TECTA-based human hereditary deafness reveal domain-specific structural phenotypes in the tectorial membrane

por Legan, P. Kevin, Goodyear, Richard J., Morín, Matías, Mencia, Angeles, Pollard, Hilary, Olavarrieta, Leticia, Korchagina, Julia, Modamio-Hoybjor, Silvia, Mayo, Fernando, Moreno, Felipe, Moreno-Pelayo, Miguel-Angel, Richardson, Guy P.
Publicado 2014

Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system

por O’Keeffe Ahern, Jonathan, Lara-Sáez, Irene, Zhou, Dezhong, Murillas, Rodolfo, Bonafont, Jose, Mencía, Ángeles, García, Marta, Manzanares, Darío, Lynch, Jennifer, Foley, Ruth, Xu, Qian, Sigen, A, Larcher, Fernando, Wang, Wenxin
Publicado 2021

Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors

por García, Marta, Bonafont, Jose, Martínez-Palacios, Jesús, Xu, Rudan, Turchiano, Giandomenico, Svensson, Stina, Thrasher, Adrian J., Larcher, Fernando, Del Rio, Marcela, Hernández-Alcoceba, Rubén, Garín, Marina I., Mencía, Ángeles, Murillas, Rodolfo
Publicado 2022

Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing

por Bonafont, Jose, Mencía, Angeles, Chacón-Solano, Esteban, Srifa, Wai, Vaidyanathan, Sriram, Romano, Rosa, Garcia, Marta, Hervás-Salcedo, Rosario, Ugalde, Laura, Duarte, Blanca, Porteus, Matthew H., Del Rio, Marcela, Larcher, Fernando, Murillas, Rodolfo
Publicado 2021

Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing

por Bonafont, Jose, Mencía, Ángeles, García, Marta, Torres, Raúl, Rodríguez, Sandra, Carretero, Marta, Chacón-Solano, Esteban, Modamio-Høybjør, Silvia, Marinas, Lucía, León, Carlos, Escamez, María J., Hausser, Ingrid, Del Río, Marcela, Murillas, Rodolfo, Larcher, Fernando
Publicado 2019