Nucleic acid products and compositions and their uses are provided. In particular, nucleic acid products are provided that modulate, interfere with, or inhibit APOC3 gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from a APOC3 gene, wherein said first nucleobase sequence is selected from the following sequences, or a portion thereof: SEQ ID NOs 1 to 39

The present disclosure provides methods for treating HBV infection using an siRNA that targets an HBV gene. In some embodiments, the method for treating HBV involves co-administration of siRNA with PEG-INFα.

The present invention discloses a targeted therapy composition and a method for suppressing breast cancer cell proliferation, migration or invasion by administrating the targeted therapy composition to a subject in need thereof. The composition comprises a type 3 epithelial membrane proteins (EMP3) targeted inhibitor. The breast cancer cells are characterized by upregulation of EMP3, EGFR, HER2, HER3, HR, or downregulation of HER4 gene expression. EMP3-targeting oligonucleotide or nuclease is administrated upon the breast cancer cells so as to degrade EMP3-coding DNA or mRNA, and to suppress breast cancer cell proliferation, migration or invasion.

The technology described herein is directed to improved chimeric molecules for, e.g, the treatment of cancer. As described herein, the inventors have developed novel chimeric aptamer-siRNA molecules (AsiCs) which demonstrate improved efficacy over existing AsiCs and which can successfully synergize in treating cancer. These AsiC's target cancer cell markers to direct therapeutic siRNA molecules specifically to cancer cells, increasing delivery efficacy and therapeutic effectiveness while reducing the potential for side effects.

Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.

The described invention provides compositions and methods for treating a fibrotic condition in a subject. The methods include administering a therapeutic amount of a pharmaceutical composition comprising synthetic extracellular vesicles (EVs) and a pharmaceutically acceptable carrier.

Provided herein are methods of treating a patient having a cancer that exhibits (i) a hemizygous loss of the TP53 gene; (ii) a hemizygous loss of the POLR2A gene; and/or (iii) a decreased level of expression of a POLR2A gene product relative to a reference (i.e., control) expression level. The methods comprise administering a therapeutically effective amount of a POLR2A inhibitor (e.g., a nucleic acid that inhibits the expression of a POLR2A protein, an amatoxin, alpha-amanitin, or alpha-amanitin conjugated to a cell targeting moiety, such as an EpCAM antibody) to a patient having or determined to have (i) a hemizygous loss of the TP53 gene; (ii) a hemizygous loss of the POLR2A gene; and/or (iii) a decreased level of expression of a POLR2A gene product relative to a reference (i.e., control) level.

An anticancer adjuvant for treating anticancer-agent-resistant cancer includes a tristetraprolin inhibitor. If resistance to DNA replication inhibitory anticancer agents is exhibited, a substance for inhibiting the activity of tristetraprolin can be included as an anticancer adjuvant so as to maximize the effects of an anticancer agent, and thus is usable in cancer treatment.

The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression in combination with one or more iron chelators and possibly other active agents, as well as therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as beta-thalassemia, sickle cell disease and transfusional iron overload or myelodysplastic syndrome, and infections and non-relapse related mortality associated with bone marrow transplantation.

This disclosure provides, e.g., novel cells having a modification (e.g., insertion or deletion) in the endogenous CD123 gene. The disclosure also provides compositions, e.g., gRNAs, that can be used to make such a modification.