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Modulating gene regulation to treat genetic disorders

Navneet Matharu, Nadav Ahituv

2020Nature Reviews Drug Discovery85 citationsDOIOpen Access PDF

Topics & Concepts

Genetic enhancementCRISPRTranslation (biology)Regulation of gene expressionComputational biologyGeneGene expressionBiologyGenome editingEnhancerEpigeneticsBioinformaticsCas9GeneticsMessenger RNACRISPR and Genetic EngineeringRNA Interference and Gene DeliveryPluripotent Stem Cells Research
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