Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X

Small molecule drugs known as modulators can treat ~90% of people with cystic fibrosis (CF), but do not work for premature termination codon variants such as W1282X (c.3846G>A). Here we evaluated two gene editing strategies, Adenine Base Editing (ABE) to correct W1282X, and Homology-Independent T...

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Detalles Bibliográficos
Autores principales: Mention, Karen, Cavusoglu-Doran, Kader, Joynt, Anya T, Santos, Lúcia, Sanz, David, Eastman, Alice C, Merlo, Christian, Langfelder-Schwind, Elinor, Scallan, Martina F, Farinha, Carlos M, Cutting, Garry R, Sharma, Neeraj, Harrison, Patrick T
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10656707/
https://www.ncbi.nlm.nih.gov/pubmed/37649273
http://dx.doi.org/10.1093/hmg/ddad143

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