l-Type amino acid transporter 1-targeting nanoparticles for antisense oligonucleotide delivery to the CNS

l-Type amino acid transporter 1 (LAT1)-specific ligands and polyion complexes are used as brain-specific targets to deliver RNA-based drugs across the blood-brain barrier. We characterized an LAT1-targeting antisense oligonucleotide (ASO)-encapsulated nanoparticle, Phe-NPs/ASO. A 25% density of phen...

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Bibliographic Details
Published in:Molecular Therapy: Nucleic Acids
Main Authors: Yu Na Lim, In Soo Ryu, Yeon-Joo Jung, Gabriel Helmlinger, Insun Kim, Hye Won Park, Hansol Kang, Jina Lee, Hyo Jin Lee, Kang Seon Lee, Ha-Na Jang, Dae-In Ha, Junghyung Park, Jinyoung Won, Kyung Seob Lim, Chang-Yeop Jeon, Hyun-Jeong Cho, Hyun Su Min, Jin-Hyeob Ryu
Format: Article
Language:English
Published: Elsevier 2024-12-01
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Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124002270