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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.sciencedirect.com/topics/neuroscience/antisense-oligonucleotide
Stanley T. Crooke, in Encyclopedia of Cancer (Second Edition), 2002. Antisense oligonucleotides are used to hybridize to a specific RNA molecule in vivo and thereby to inhibit its subsequent use; in most cases, the target RNA is a messenger RNA (mRNA), which cannot then be translated into a protein. Once the gene sequence of interest is known, an antisense oligonucleotide with the ...
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.nature.com/articles/nrneurol.2017.148
Dec 01, 2017 · An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol. 12 , …Author: C Rinaldi, Wood Mja.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3381599/
Traditionally, systemic delivery of oligonucleotide drugs has been performed by intravenous injection of either naked molecules or within delivery systems, but nonspecific accumulation in various tissues has favored local delivery routes such as to the lungs so as to ensure direct access to the target site.Author: Jørgen Kjems, Kenneth A Howard
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://ng.neurology.org/content/5/2/e323
Apr 01, 2019 · There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine. Indeed, existing pipelines for the development of ASO therapies for spinocerebellar ataxias, Huntington disease, …Author: Daniel R. Scoles, Eric V. Minikel, Stefan M. Pulst
http://www.healthtech.com/oligonucleotide/
Oligonucleotide Therapeutics and Delivery Conference being held April 5-6, 2016 in Cambridge, Massachusetts Oligonucleotide–based therapeutics have long been considered as forming the third major drug development platform, specifically focused on modulating gene expression by targeting RNA transcripts or the genome itself. A key distinguishing attribute of utilizing nucleic acids as ...
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