The present invention relates to novel cationic lipids, transfection
agents, microparticles, nanoparticles, and short interfering nucleic acid
(siNA) molecules. The invention also features compositions, and methods
of use for the study, diagnosis, and treatment of traits, diseases and
conditions that respond to the modulation of gene expression and/or
activity in a subject or organism. Specifically, the invention relates to
novel cationic lipids, microparticles, nanoparticles and transfection
agents that effectively transfect or deliver biologically active
molecules, such as antibodies (e.g., monoclonal, chimeric, humanized
etc.), cholesterol, hormones, antivirals, peptides, proteins,
chemotherapeutics, small molecules, vitamins, co-factors, nucleosides,
nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic
acids, triplex forming oligonucleotides, 2,5-A chimeras, dsRNA,
allozymes, aptamers, decoys and analogs thereof, and small nucleic acid
molecules, such as short interfering nucleic acid (siNA), short
interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA),
short hairpin RNA (shRNA), and RNAi inhibitor molecules, to relevant
cells and/or tissues, such as in a subject or organism. Such novel
cationic lipids, microparticles, nanoparticles and transfection agents
are useful, for example, in providing compositions to prevent, inhibit,
or treat diseases, conditions, or traits in a cell, subject or organism.
The compositions described herein are generally referred to as formulated
molecular compositions (FMC) or lipid nanoparticles (LNP).