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

• Главные авторы: Irene Vázquez-Domínguez (Автор), Mert Öktem (Автор), Florian A. Winkelaar (Автор), Thai Hoang Nguyen (Автор), Anita D.M. Hoogendoorn (Автор), Eleonora Roschi (Автор), Galuh D.N. Astuti (Автор), Raoul Timmermans (Автор), Nuria Suárez-Herrera (Автор), Ilaria Bruno (Автор), Albert Ruiz-Llombart (Автор), Joseph Brealey (Автор), Olivier G. de Jong (Автор), Rob W.J. Collin (Автор), Enrico Mastrobattista (Автор), Alejandro Garanto (Автор)
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• Опубликовано: Elsevier, 2024-12-01T00:00:00Z.
• Предметы: article
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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...