Efficient genome editing in the non-human primate brain using programmable extracellular vesicles

Abstract excerpt

<h4>ABSTRACT</h4> In vivo genome editing holds transformative potential for treating genetic disease, yet the absence of safe, efficient and scalable delivery systems remains a major barrier to clinical translation. While progress has been made in ex vivo and liver-directed editing, delivery to extrahepatic tissues, particularly the central nervous system (CNS), remains a fundamental challenge, limiting therapeu...

Topics

Open a Topic to create a Post that cites this publication.

• CRISPR-Cas Systems
• Extracellular Vesicles
• Extracellular vesicles in disease
• Gene Editing
• Gene Transfer Techniques
• Mutation
• Parvovirus B19 Infection Studies
• RNA

Identifiers and source

Literature Corpus work

71fcba22-41cd-51ed-9842-451c0126dd2f

DOI

10.64898/2026.08.05.741964