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

• Main Authors: Amy E. Byrnes (Author), Sara L. Dominguez (Author), Chun-Wan Yen (Author), Benjamin I. Laufer (Author), Oded Foreman (Author), Mike Reichelt (Author), Han Lin (Author), Meredith Sagolla (Author), Kathy Hötzel (Author), Hai Ngu (Author), Christoffer Soendergaard (Author), Alberto Estevez (Author), Hsiu-Chao Lin (Author), Alexandre Goyon (Author), Juan Bian (Author), Jessica Lin (Author), Flora I. Hinz (Author), Brad A. Friedman (Author), Amy Easton (Author), Casper C. Hoogenraad (Author)
• Format: Book
• Published: Elsevier, 2023-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...