Haplotype editing with CRISPR-Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL

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Authors: Poorvi H. Dua, Bazilco M.J. Simon, Chiara B.E. Marley, Carissa M. Feliciano, Hannah L. Watry, Quinn T. Cowan, Dylan Steury, Abin Abraham, Erin N. Gilbertson, Grace D. Ramey, John A. Capra, Bruce R. Conklin, and  Luke M. Judge

Molecular Therapy, 19 November 2025

 

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