Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4

Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4

Deep-intronic (DI) variants represent approximately 10%–12% of disease-causing genetic defects in ABCA4-associated Stargardt disease (STGD1). Although many of these DI variants are amenable to antisense oligonucleotide-based splicing-modulation therapy, no treatment is currently available. These mol...

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Bibliographic Details
Main Authors: Irene Vázquez-Domínguez, Mert Öktem, Florian A. Winkelaar, Thai Hoang Nguyen, Anita D.M. Hoogendoorn, Eleonora Roschi, Galuh D.N. Astuti, Raoul Timmermans, Nuria Suárez-Herrera, Ilaria Bruno, Albert Ruiz-Llombart, Joseph Brealey, Olivier G. de Jong, Rob W.J. Collin, Enrico Mastrobattista, Alejandro Garanto
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
peptide-mediated delivery
CRISPR-Cas9 genome editing
lipopeptide
intron removal
ABCA4 deep-intronic variants
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124002324

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http://www.sciencedirect.com/science/article/pii/S2162253124002324

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