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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Detalles Bibliográficos
Autores principales: Byrnes, Amy E., Dominguez, Sara L., Yen, Chun-Wan, Laufer, Benjamin I., Foreman, Oded, Reichelt, Mike, Lin, Han, Sagolla, Meredith, Hötzel, Kathy, Ngu, Hai, Soendergaard, Christoffer, Estevez, Alberto, Lin, Hsiu-Chao, Goyon, Alexandre, Bian, Juan, Lin, Jessica, Hinz, Flora I., Friedman, Brad A., Easton, Amy, Hoogenraad, Casper C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10280097/
https://www.ncbi.nlm.nih.gov/pubmed/37346977
http://dx.doi.org/10.1016/j.omtn.2023.05.005

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