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

• Main Authors: Irene Vázquez-Domínguez (Author), Mert Öktem (Author), Florian A. Winkelaar (Author), Thai Hoang Nguyen (Author), Anita D.M. Hoogendoorn (Author), Eleonora Roschi (Author), Galuh D.N. Astuti (Author), Raoul Timmermans (Author), Nuria Suárez-Herrera (Author), Ilaria Bruno (Author), Albert Ruiz-Llombart (Author), Joseph Brealey (Author), Olivier G. de Jong (Author), Rob W.J. Collin (Author), Enrico Mastrobattista (Author), Alejandro Garanto (Author)
• Format: Book
• Published: Elsevier, 2024-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...