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https://www.genengnews.com/magazine/114/delivery-approaches-for-rnai-based-drugs/
Jun 01, 2009 · Two of the most popular methods of siRNA delivery are nanoparticle- and liposome-based systems. The liposome-based systems have, up to now, been associated with liver-targeted pharmacokinetics, a phenomenon that restricts the therapeutic potential of RNAi to common diseases of the liver such as hepatitis,...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3378126/
Mar 16, 2009 · The key challenge for RNAi application: delivery. As with the older oligonucleotide technologies using antisense DNA or catalytic RNAs, delivery to the target cells and tissues is a major challenge for RNAi based drugs. Thus, delivery of siRNAs to specific cells needs to be addressed for safe and effective in vivo RNAi drug applications. The negative charge of siRNAs as well as their size …Author: Katrin Tiemann, John J. Rossi
https://genomemedicine.biomedcentral.com/articles/10.1186/s13073-017-0450-0
Jun 27, 2017 · This research is finally beginning to bear fruit as the first RNA drugs gain FDA approval and more advance to the final phases of clinical trials. Furthermore, the recent advent of CRISPR, an RNA-guided gene-editing technology, as well as new strides in the delivery of messenger RNA transcribed in vitro,...Author: James C. Kaczmarek, Piotr S. Kowalski, Daniel G. Anderson
https://phys.org/news/2018-08-rna-drug.html
Aug 17, 2018 · There is also continued research and development of RNA-based therapies to treat disorders caused by changes in the cell triggered by a close cousin of siRNA, called microRNA. RNA drugs offer a new weapon in the arsenal to treat disease …Author: Thomas Schmittgen
https://www.nature.com/articles/s41573-019-0017-4
Mar 07, 2019 · Small double-stranded RNA molecules can efficiently trigger RNAi silencing of specific genes, but their therapeutic use has faced numerous challenges involving safety and potency. However, August 2018 marked a new era for the field, with the US Food and Drug Administration approving patisiran, the first RNAi-based drug.Author: Ryan L. Setten, Ryan L. Setten, John J. Rossi, John J. Rossi, Si-ping Han, Si-ping Han
https://www.ddw-online.com/therapeutics/p322575-current-trends-in-rna-therapeutics-development.html
However, delivery across the lipid bilayer remains a significant challenge and approaches to enhance endosomal escape of RNA drugs are required. To date, four RNA-based drugs – Macugen, Exondys 51, Spinraza and Onpattro – have successfully made it through to market and several other RNA agents are currently in clinical programmes.
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