Provided a novel miR-96-5p inhibitor that is more effective than an antisense oligonucleotide against miR-96-5p, and a novel pharmaceutical composition comprising the same. The miR-96-5p inhibitor comprises a peptide nucleic acid moiety comprising a nucleotide sequence complementary to miR-96-5p.

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Collagen gene, in particular, by targeting natural antisense polynucleotides of a Collagen gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Collagen genes.

The present invention provides the peptide nucleic acid derivative which targets 5′ splice site of the human ACC2 pre-mRNA “exon 12”. The peptide nucleic acid derivatives in the present invention strongly induce splice variants of the human ACC2 mRNA in cell and are very useful to treat conditions or disorders of skin aging associated with the human ACC2 protein.

The present inventions generally relate to site-specific delivery of nucleic acid modifiers and includes novel DNA-binding proteins and effectors that can be rapidly programmed to make site-specific DNA modifications. The present inventions also provide a synthetic all-in-one genome editor (SAGE) systems comprising designer DNA sequence readers and a set of small molecules that induce double-strand breaks, enhance cellular permeability, inhibit NHEJ and activate HDR, as well as methods of using and delivering such systems.

Described herein are methods and compositions related to the modulation of progranulin expression or activity in the brain for the treatment of neurodegenerative diseases.

The present invention relates to a pharmaceutical composition comprising an agent inhibiting gene expression or protein activity of osteopontin as an active ingredient for preventing or treating neurodegenerative disease. According to the present invention, there is an effect of suppressing amyloid beta (Aβ)-induced neuronal cell death by downregulating the expression or activity of osteopontin. In addition, an inhibitor against the expression or activity of osteopontin decreases a level of pro-inflammatory proteins and conversely, increases a level of anti-inflammatory proteins. Therefore, it is expected that the present invention can be advantageously used as a therapeutic agent for various neurodegenerative diseases including Alzheimer's disease.

The present disclosure provides, inter alia, improved PNA agents, compositions comprising the same, and methods for treating diseases such as cancers by using such agents and/or compositions. Methods for reducing expression of a gene in a cell are also provided.

The present disclosure relates to the technical field of genetic engineering, in particular to a siRNA of an angiopoietin like 3 (ANGPTL3) and a use thereof. The inventor of the present disclosure targets to ANGPTL3 by designing an appropriate specific small interfering RNA sequence and a siRNA conjugate, and reduce the expression of an ANGPTL3 protein by degrading a transcript of an ANGPTL3 gene in a cell. Therefore, the siRNA provided in the present disclosure may be used to prevent and/or treat a dyslipidemia disease.

Disclosed herein include methods and compositions for selectively amplifying and/or extending nucleic acid target molecules in a sample. The methods and compositions can, for example, reduce the amplification and/or extension of undesirable nucleic acid species in the sample, and/or allow selective removal of undesirable nucleic acid species in the sample.

Provided are peptide nucleic acid derivatives targeting a part of the human HIF-1α pre-mRNA. The peptide nucleic acid derivatives potently induce exon skipping to yield splice variants of HIF-1α mRNA in cells, and are useful to treat indications or conditions involving the overexpression of HIF-1α.