Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo**
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Machine scores (provisional)
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- Teacher spread
- 0.242 · how far apart the two teachers sit on this one work
- Validation status
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Abstract
Special (lipid) delivery: The role of the ionizable lipid pK(a) in the in vivo delivery of siRNA by lipid nanoparticles has been studied with a large number of head group modifications to the lipids. A tight correlation between the lipid pK(a) value and silencing of the mouse FVII gene (FVII ED(50) ) was found, with an optimal pK(a) range of 6.2-6.5. The most potent cationic lipid from this study has ED(50) levels around 0.005 mg kg(-1) in mice and less than 0.03 mg kg(-1) in non-human primates.
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The record
- Venue
- Angewandte Chemie International Edition
- Topic
- RNA Interference and Gene Delivery
- Field
- Biochemistry, Genetics and Molecular Biology
- Canadian institutions
- University of British ColumbiaEncana (Canada)
- Funders
- Canadian Institutes of Health Research
- Keywords
- Gene silencingIn vivoPotencyChemistryIn vitroGene deliveryLipid metabolismCationic polymerizationBiochemistryTransfectionMolecular biologyGeneBiophysicsBiologyGeneticsOrganic chemistry
- Has abstract in OpenAlex
- yes