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https://onlinelibrary.wiley.com/doi/abs/10.1002/anie.201914751
Jan 29, 2020 · Systemic Brain Delivery of Antisense Oligonucleotides across the Blood–Brain Barrier with a Glucose‐Coated Polymeric Nanocarrier. Dr. Hyun Su Min. Department of Materials Engineering, Graduate School of Engineering, The University of Tokyo, …Author: Hyun Su Min, Hyun Jin Kim, Mitsuru Naito, Satomi Ogura, Kazuko Toh, Kotaro Hayashi, Beob Soo Kim, Sh...
https://www.sciencedirect.com/science/article/pii/S0169409X15000435
2.1. Peripheral delivery. Efforts are ongoing to deliver antisense oligonucleotides to the nervous system via the systemic route. In general, when a drug is administered systemically, a fraction will be bound to proteins (e.g. serum albumin, lipoprotein etc.) and a fraction will be unbound.Author: Melvin M. Evers, Lodewijk J.A. Toonen, Willeke M.C. van Roon-Mom
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2475625/
Jun 16, 2008 · The potential use of antisense and siRNA oligonucleotides as therapeutic agents has elicited a great deal of interest. However, a major issue for oligonucleotide-based therapeutics involves effective intracellular delivery of the active molecules.Author: Rudy Juliano, Md. Rowshon Alam, Vidula Dixit, Hyumin Kang
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6248909/
Apr 16, 2018 · Exon-skipping antisense oligonucleotides are effective treatments for genetic diseases, yet exon-skipping activity requires that these macromolecules reach the nucleus. While cell-penetrating peptides can improve delivery, proteolytic instability often ...Author: Justin M. Wolfe, Colin M. Fadzen, Rebecca L. Holden, Monica Yao, Gunnar J. Hanson, Bradley L. Pentel...
https://www.sciencedirect.com/science/article/pii/S2162253120300536
The efficient delivery of antisense oligonucleotides (ASOs) to the targeted cells and organs remains a challenge, in particular, in vivo.Here, we investigated the ability of a library of biodegradable lipid nanoparticles (LNPs) in delivering ASO to both cultured human cells and animal models.Author: Liu Yang, Feihe Ma, Fang Liu, Jinjin Chen, Xuewei Zhao, Qiaobing Xu
https://www.nature.com/articles/nrneurol.2017.148
Dec 01, 2017 · Falzarano, M. S., Passarelli, C. & Ferlini, A. Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy.Author: C Rinaldi, Wood Mja.
https://advances.sciencemag.org/content/4/10/eaat3386.full
Oct 01, 2018 · Antisense oligonucleotide (ASO) silencing of the expression of disease-associated genes is an attractive novel therapeutic approach, but treatments are limited by the ability to deliver ASOs to cells and tissues. Following systemic administration, ASOs preferentially accumulate in liver and kidney. Among the cell types refractory to ASO uptake is the pancreatic insulin-secreting β-cell. Here ...Author: C. Ämmälä, W. J. Drury, L. Knerr, I. Ahlstedt, P. Stillemark-Billton, C. Wennberg-Huldt, E.-M. Ander...
https://academic.oup.com/nar/article/42/13/8796/1283685
Jul 03, 2014 · In this report, we used a previously identified ASGPR ligand (GN3), which has been adapted for attachment to oligonucleotides , to increase delivery of PS gapmer ASOs into hepatocytes. GN3-conjugated ASOs showed high affinity for mouse ASGPR, which results in enhanced ASO delivery to hepatocytes versus np cells.Author: Thazha P. Prakash, Mark J. Graham, Jinghua Yu, Rick Carty, Audrey Low, Alfred Chappell, Karsten Schm...
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