Article
ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation.
Scientific reports - 16 Nov 2022
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
Abstract excerpt
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. Adenine base editors (ABEs) convert A-T base pairs to G-C base pairs without requiring double-stranded DNA breaks or...
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