TY  - JOUR
AU  - Bonafont, Jose
AU  - Mencía, Ángeles
AU  - García, Marta
AU  - Torres-Ruiz, Raul
AU  - Rodriguez Perales, Sandra
AU  - Carretero, Marta
AU  - Chacón-Solano, Esteban
AU  - Modamio-Høybjør, Silvia
AU  - Marinas, Lucía
AU  - León, Carlos
AU  - Escamez, María J
AU  - Hausser, Ingrid
AU  - Del Río, Marcela
AU  - Murillas, Rodolfo
AU  - Larcher, Fernando
PY  - 2019
DO  - 10.1016/j.ymthe.2019.03.007
SN  - 15250016
UR  - http://hdl.handle.net/20.500.12105/7611
AB  - Gene editing constitutes a novel approach for precisely correcting disease-causing gene mutations. Frameshift mutations in COL7A1 causing recessive dystrophic epidermolysis bullosa are amenable to open reading frame restoration by non-homologous end...
LA  - eng
PB  - Elsevier
KW  - CRISPR/Cas9
KW  - Epidermal stem cells
KW  - Epidermolysis bullosa
KW  - Gene therapy
TI  - Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing
TY  - journal article
ER  -