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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2698461/
In one example, RNAi delivery vectors that target epidermal growth factor receptor (EGFR), which are expressed by a majority of aggressive brain tumors, have been encapsulated in PEGylated immunoliposomes (poly-ethylene-glycol ‘encapsulated’ liposomes) and targeted in an orthotopic brain tumor animal model.Author: Saroj P Mathupala
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3572210/
Mar 30, 2007 · The limiting factor in in vivo RNA interference (RNAi) is delivery. Drug delivery methods that are effective in cell culture may not be practical in vivo for intravenous RNAi applications. Nucleic acid drugs are highly charged and do not cross cell membranes by free diffusion.Author: William M. Pardridge
https://www.ncbi.nlm.nih.gov/pubmed/17434235
Mar 30, 2007 · Therefore, the in vivo delivery of RNAi therapeutics must use targeting technology that enables the RNAi therapeutic to traverse biological membrane barriers in vivo. For RNAi of the brain, the nucleic acid-based drug must first cross the brain capillary endothelial wall, which forms the blood-brain barrier (BBB) in vivo, and then traverses the brain cell plasma membrane.Author: William M. Pardridge
https://www.sciencedirect.com/science/article/abs/pii/S0169409X07000221
Weekly, intravenous RNAi with PILs enables a 90% knockdown of the human epidermal growth factor receptor, which results in a 90% increase in survival time in mice with intra-cranial brain cancer. Similar to the delivery of antisense agents, short interfering RNAi (siRNA) duplexes can be delivered with the combined use of targeting MAb's and ...Author: William M. Pardridge
https://www.sciencedirect.com/science/article/pii/S0169409X07000221
For RNAi of the brain, the nucleic acid-based drug must first cross the brain capillary endothelial wall, which forms the blood–brain barrier (BBB) in vivo, and then traverses the brain cell plasma membrane. Similar to the delivery of non-viral gene therapies, plasmid DNA encoding for short hairpin RNA...Author: William M. Pardridge
https://www.alnylam.com/2019/03/29/pre-clinical-data-delivery-rnai-therapeutics-cns-eye/
Mar 29, 2019 · We presented new pre-clinical data on our efforts in CNS and ocular delivery of novel siRNA conjugates in rats and non-human primates at the sixth annual Cold Spring Harbor Conference on RNA and Oligonucleotide Therapeutics, held March 27 to 30 in Cold Spring Harbor, NY.
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