Article
Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor.
Molecular therapy : the journal of the American Society of Gene Therapy - 7 Jun 2023
Amistadi Simone, Maule Giulia, Ciciani Matteo, Ensinck Marjolein M, De Keersmaecker Liesbeth, Ramalho Anabela S, Guidone Daniela, Buccirossi Martina, Galietta Luis J V, Carlon Marianne S, Cereseto Anna
Abstract excerpt
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. The 2789+5G>A CFTR mutation is a quite frequent defect causing an aberrant splicing and a non-functional CFTR protein. Here we used a CRISPR adenine base editing (ABE) approach to correct the mutation in the absence of DNA double-strand breaks (DSB). To select the strategy, we developed a...
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