Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids

Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids

Summary: Cystic fibrosis (CF) is a life-shortening autosomal recessive disease, caused by loss-of-function mutations that affect the CF transmembrane conductance regulator (CFTR) anion channel. G542X is the second-most common CF-causing variant, and it does not respond to current CFTR modulator drug...

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Bibliographic Details
Main Authors: Lucia Nicosia, Iwona Pranke, Roberta V. Latorre, Joss B. Murray, Lisa Lonetti, Kader Cavusoglu-Doran, Elise Dreano, James P. Costello, Michael Carroll, Paola Melotti, Claudio Sorio, Isabelle Sermet-Gaudelus, Martina F. Scallan, Patrick T. Harrison
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:iScience
Subjects:
Health sciences
Clinical genetics
Cellular therapy
Genetic engineering
Online Access:http://www.sciencedirect.com/science/article/pii/S2589004225002391
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http://www.sciencedirect.com/science/article/pii/S2589004225002391

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