Abstract: The instant disclosure relates to nucleic acid-lipid particles having 1,2-dioleoyl-3-trimethylammonium-propane (DOTAP), which preferentially localize and deliver associated cargoes to the lung and various lung tissues, as well as to tissues to which such particles are directly injected. The instant disclosure provides compositions comprising such lipid particles, optionally in association with a therapeutic agent (e.g., a therapeutic mRNA and/or nucleic acid controller system), as well as methods and kits for delivering a lipid particle-associated therapeutic agent and/or treating a disease or disorder, e.g., a lung disease or disorder, in a subject, using the lipid particle compositions provided herein.

Abstract: The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of an apolipoprotein B (APOB) gene by targeting an APOB expression control region and methods of use thereof for treating an APOB associated disorder, e.g., hypercholesterolemia.

Abstract: One aspect of the invention provides a method of separating long polynucleotides from a composition. The method includes: introducing a composition including one or more long polynucleotides into a container including at least one boundary defined by a filter comprising a plurality of pores, wherein the pores have a smaller cross-sectional dimension than specified for the long polynucleotide's molecular weight; and applying elevated hydraulic pressure to the composition, thereby causing at least some of the one or more long polynucleotides to pass through the pores.