The present disclosure provides catanionic surfactant vesicles (SVs). The vesicles may be functionalized on their outer leaflet such that they may be biologically active. The vesicles may encapsulate (at least partially in the lumen and/or at least partially in the leaflet) one or more small molecules, one or more RNAs, one or more DNAs, and/or one or more proteins/peptides. Also provided are compositions comprising the vesicles (e.g., vaccine compositions comprising the vesicles) and methods of making and using the same.

The present invention relates to a conjugate for delivery of biomolecules using a cationic macromolecule as a linker between the biomolecule and biomarker targeting moiety, and the process of making the conjugate.

The invention relates to a long non-coding RNA (LETN) useful as a diagnostic and therapeutic target for cancer. In particular, the invention discloses that lncRNA RP11-196G18.22 (LETN) is overexpressed in cancer cells, and such overexpression can promote the proliferation of cancer cells and are associated with short prognostic survival time in cancer patients. Reducing the expression of this lncRNA results in the inhibition of cancer cell growth, and thus inhibiting the expression of this lncRNA represents a new strategy for cancer therapy

The present invention relates to an anti-tumor adenovirus and an anticancer composition comprising same. Double-stranded siRNA of the present invention simultaneously inhibits the expression of a first nucleic acid and a second nucleic acid, thus promoting the death of cancer cells, exhibits more remarkable anticancer activity as compared to co-treatment of respective siRNAs, has a synergistic effect of improving cancer cell death in combined treatment with an anticancer agent. The adenovirus comprising a shRNA-encoding expression cassette expressing the double-stranded siRNA, and a hTERT promoter evades immune responses in the body and is specifically delivered to cancer cells, thus having a systemic therapeutic effect, can be locally delivered, has excellent selectivity, and exhibits a significant anticancer effect even in minimally invasive treatment, and thus, the adenovirus can be effectively used as an anticancer composition or an anticancer adjuvant in various carcinomas.

This application pertains to a hairpin nucleic acid molecule capable of modulating expression of a target gene and a use thereof. A nucleic acid molecule according to an embodiment can modulate expression of a target gene in a specific manner for cells in which a miRNA hybridizable therewith is present, finding advantageous applications in compositions for regulating expression of a target gene or pharmaceutical compositions for treating diseases.

The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

Provided is a method for high-efficiently reading through a nonsense mutation site in a pathogenic gene in a monogenic hereditary disease and restoring the normal structure and function of a mutant protein, by using a genetic code expanded non-natural amino acid system. By modifying a tRNA of Methanosarcina barkeri (tRNAPyl), an all-new UAA and UGA encoded non-natural amino acid system that has high read-through efficiency is obtained, and the range of using the orthogonal pair of tRNAPyl and pyrrolysyl-tRNA synthetase (PylRS) is expanded. A plasmid mimicking the endogenous premature termination codon is constructed, so as to evaluate the efficiency of reading through the endogenous premature termination codon. Also provided is a system mainly comprising pathogenic genes of monogenic hereditary diseases and tumor inhibitory genes in tumor cells.

This disclosure relates to oligonucleotides, compositions and methods useful for reducing CTNNB1 expression, particularly in hepatocytes, for the treatment of bile duct paucity-associated conditions. Disclosed oligonucleotides for the reduction of CTNNB1 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of CTNNB1 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver.

The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

The present invention relates to a COPS3 inhibitor for use in treatment of an HBV infection, in particular a chronic HBV infection. The invention in particular relates to the use of COPS3 inhibitors for destabilizing cccDNA, such as HBV cccDNA. The invention also relates to nucleic acid molecules which are complementary to COPS3 and capable of reducing the level of a COPS3 mRNA. Also comprised in the present invention is a pharmaceutical composition and its use in the treatment of a HBV infection.