Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection

Antisense oligonucleotide (ASO) therapeutics are being investigated for a broad range of neurological diseases. While ASOs have been effective in the clinic, improving productive ASO internalization into target cells remains a key area of focus in the field. Here, we investigated how the delivery of...

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Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Amy E. Byrnes, Sara L. Dominguez, Chun-Wan Yen, Benjamin I. Laufer, Oded Foreman, Mike Reichelt, Han Lin, Meredith Sagolla, Kathy Hötzel, Hai Ngu, Christoffer Soendergaard, Alberto Estevez, Hsiu-Chao Lin, Alexandre Goyon, Juan Bian, Jessica Lin, Flora I. Hinz, Brad A. Friedman, Amy Easton, Casper C. Hoogenraad
Format:	Article
Language:	English
Published:	Elsevier 2023-06-01
Subjects:	MT: Delivery Strategies antisense oligonucleotide delivery gymnosis lipid nanoparticles central nervous system
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253123001221