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

Genome-editing technologies that enable the introduction of precise changes in DNA sequences have the potential to lead to a new class of treatments for genetic diseases. Epidermolysis bullosa (EB) is a group of rare genetic disorders characterized by extreme skin fragility. The recessive dystrophic...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2021
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8178438/
https://ncbi.nlm.nih.gov/pubmed/33609734
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2021.02.019
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