Adenine Base Editing with Engineered Virus-Like Particles Rescues the CFTR Mutation G542X in Patient-Derived Intestinal Organoids

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

iScience（2025）

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Key words

Health sciences,Clinical genetics,Cellular therapy,Genetic engineering

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