| abstract |
The present invention relates to novel cationic lipids, transfection agents, microparticles, nanoparticles, and small interfering nucleic acid (siNA) molecules. The invention also features compositions and methods used for the study, diagnosis, and treatment of traits, diseases and conditions that respond to modulation of gene expression and / or activity in a patient or organism. Specifically, the present invention relates to biologically active molecules such as antibodies (eg, monoclonal antibodies, chimeric antibodies, humanized antibodies, etc.), cholesterol, hormones, antiviral agents, peptides, proteins, chemotherapeutic agents, Small molecule, vitamin, cofactor, nucleoside, nucleotide, oligonucleotide, enzymatic nucleic acid, antisense nucleic acid, triplex forming oligonucleotide, 2,5-A chimera, dsRNA, allozyme, aptamer, decoy and analogs thereof, And small nucleic acid molecules, such as small interfering nucleic acids (siNA), small interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), short hairpin RNA (shRNA), and RNAi inhibitor molecules For example, effective transfer to relevant cells and / or tissues of a patient or organism. The novel cationic lipids for transfected or delivery, microparticles, relates nanoparticles and transfection agents. Such novel cationic lipids, microparticles, nanoparticles and transfection agents provide compositions for preventing, inhibiting or treating diseases, conditions, or traits, for example, in cells, patients or organisms. Useful for. The compositions described herein are commonly referred to as formulated molecular compositions (FMC) or lipid nanoparticles (LNP). |