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The therapeutic implications of all-in-one AAV-delivered epigenome-editing platform in neurodegenerative disorders

23 Aug 2024

Abstract excerpt

Safely and efficiently controlling gene expression is a long-standing goal of biomedical research, and CRISPR/Cas system can be harnessed to create powerful tools for epigenetic editing. Adeno-associated-viruses (AAVs) represent the delivery vehicle of choice for therapeutic platform. However, their small packaging capacity isn’t suitable for large constructs including most CRISPR/dCas9-effector vectors. Thus,...

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PMID: 39179542
DOI: 10.1038/s41467-024-50515-6

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