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Optimization of S. aureus dCas9 and CRISPRi Elements for a Single Adeno-Associated Virus that Targets an Endogenous Gene

Jon R. Backstrom, Jinsong Sheng, Michael Cai Wang, Alexandra Bernardo‐Colón, Tonia S. Rex

2020Molecular Therapy — Methods & Clinical Development21 citationsDOIOpen Access PDF

Abstract

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Topics & Concepts

Adeno-associated virusBiologyProprotein convertaseGeneMolecular biologyGuide RNAPromoterGene expressionCell biologyVirologyVector (molecular biology)GeneticsCRISPRCas9Recombinant DNABiochemistryLDL receptorCholesterolLipoproteinCRISPR and Genetic EngineeringVirus-based gene therapy researchViral Infections and Immunology Research