Article

Advancing Liver Gene Therapy: Enhanced Transduction with GalNAc-Bioconjugated rAAV Capsids

2024-07-25

Abstract excerpt

This study investigates novel approaches to improve targeted gene delivery to the liver, a crucial organ for metabolic processes that faces vulnerabilities from various pathologies. Adeno-associated virus (AAV)-based gene therapy has emerged as a promising approach for liver targeting, with numerous investigational avenues. However, administration of high doses of AAV vectors present safety concerns, often requiri...

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Literature Corpus work: 88ab088d-7535-5dda-b9fe-0e740751a509
DOI: 10.1101/2024.07.25.605063

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