Acuitas Therapeutics (Canada)
ORCID: 0000-0002-4083-5251Publishes on RNA Interference and Gene Delivery, Immunotherapy and Immune Responses, SARS-CoV-2 and COVID-19 Research. 190 papers and 21.8k citations.
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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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