The present invention provides compositions comprising therapeutic nucleic acids such as siRNA that target ApoC3 and ANGPTL3 expression, lipid particles comprising one or more (e.g., a combination) of the therapeutic nucleic acids, and methods of delivering and/or administering the lipid particles (e.g., for treating hypertriglyceridemia in humans).

Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of α1-antitrypsin (AAT), such as seen in subjects having α1-antitrypsin deficiency (AATD), are provided.

Provided are compositions and methods for using the same. In some embodiments, the compositions include an EPELN encapsulating and/or having associated therewith an active agent and a plasma membrane derived from a tumor and/or cancer cell coating the EPELN. In some embodiments, the active agent is a therapeutic agent or an immune response modifier, and in some embodiments the plasma membrane has one or more tumor-associated and/or cancer-associated antigens. Also provided are methods for using the compositions for treating tumors and/or cancers, inducing anti-tumor and/or anti-cancer immune responses, activating antigen-presenting cells, targeting CD11c dendritic cells, and preventing or reducing metastasis.

Methods and agents that target nanog or Oct4 expression or activity for treating or preventing cancer are disclosed. Alternative methods involve diagnosing cancer stage or type by identifying presence of cancer cells expressing nanog or Oct4. Also, disclosed are method of treating coronavirus infection that involves administering antiviral knockdown agents, such as oligonucleotide-based inhibitors.

Provided are compositions and methods useful in regenerating damaged tissue, especially cardiac tissue, by delivering to the site of injury an miRNA that can reduce, for example, the expression of phosphatase and tensin homolog (PTEN). The compositions and methods of the disclosure may be generally applied to deliver an miRNA to a cell or tissue such as, but not limited to, a neuron, a smooth muscle cell, or a tumor cell. The compositions comprise a transmembrane carrier peptide conjugated, optionally by a linker, to an oligonucleotide complementary to an miRNA. The carrier peptide facilitates the entry of the miRNA into cells and for delivery to a tissue of an animal or human may be mixed with an extracellular matrix-derived hydrogel carrier.

Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of α1-antitrypsin (AAT), such as seen in subjects having α1-antitrypsin deficiency (AATD), are provided.

Provided herein are MAPT RNAi agents and compositions comprising a MAPT RNAi agent. Also provided herein are methods of using the MAPT RNAi agents or compositions comprising a MAPT RNAi agent for reducing MAPT expression and/or treating tauopathy in a subject.

Disclosed herein is a formulation comprising an extracellular vesicle (EV), and a therapeutic active agent induced or embedded in the EV. According to preferred embodiments of the present disclosure, the EV is isolated from umbilical cord mesenchymal stem cells, and the active agent may be a growth factor, an immune-modulating agent, a small molecule, an siRNA, cDNA or a plant ingredient; for example, curcumin. Also disclosed herein are methods for producing the present formulation, and uses of the present formulation in the treatment of various diseases.

The present disclosure generally relates to metal-organic framework nanoparticles containing terminal phosphate-modified oligonucleotides, methods for making the same, and methods of using the same.

Described herein are lipid nanoparticles comprising cationic lipids and other lipids and also comprising engineered nucleases facilitate transfer of nucleic acids to cells.