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Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4 |
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Titel: |
Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4 |
Auteur: |
Vázquez-Domínguez, Irene Öktem, Mert Winkelaar, Florian A. Nguyen, Thai Hoang Hoogendoorn, Anita D.M. Roschi, Eleonora Astuti, Galuh D.N. Timmermans, Raoul Suárez-Herrera, Nuria Bruno, Ilaria Ruiz-Llombart, Albert Brealey, Joseph de Jong, Olivier G. Collin, Rob W.J. Mastrobattista, Enrico Garanto, Alejandro |
Verschenen in: |
Molecular therapy - nucleic acids |
Paginering: |
Jaargang 35 () nr. 4 pagina's p. |
Jaar: |
2024 |
Inhoud: |
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Uitgever: |
The Author(s) |
Bronbestand: |
Elektronische Wetenschappelijke Tijdschriften |
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