ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation

Base editing introduces precise single-nucleotide edits in genomic DNA and has the potential to treat genetic diseases such as the blistering skin disease recessive dystrophic epidermolysis bullosa (RDEB), which is characterized by mutations in the COL7A1 gene and type VII collagen (C7) deficiency....

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Detalles Bibliográficos
Autores principales: Sheriff, Adam, Guri, Ina, Zebrowska, Paulina, Llopis-Hernandez, Virginia, Brooks, Imogen R., Tekkela, Stavroula, Subramaniam, Kavita, Gebrezgabher, Ruta, Naso, Gaetano, Petrova, Anastasia, Balon, Katarzyna, Onoufriadis, Alexandros, Kujawa, Dorota, Kotulska, Martyna, Newby, Gregory, Łaczmański, Łukasz, Liu, David R., McGrath, John A., Jacków, Joanna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9666996/
https://www.ncbi.nlm.nih.gov/pubmed/36385635
http://dx.doi.org/10.1038/s41598-022-24184-8

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