In the present specification, on the basis of the correlation between prospero homeobox protein 1 (PROX1) and telomerase reverse transcriptase (TERT), a composition for regulating expression of TERT, a method for screening a TERT expression regulator, a composition for diagnosing a TERT expression status, a diagnostic kit, a method for providing information for diagnosis, or a method for providing information for cancer diagnosis are disclosed. Specifically, in one aspect, the PROX1 of the present disclosure may bind to a TERT promoter, in particular a mutant TERT promoter in which base substitution occurs at the -124 or -146 bp position to regulate the expression of TERT, and the expression of TERT in non-hepatitis B virus-associated liver cancer can be inhibited specifically among liver cancers.

The present invention relates to a composition for preventing or treating skin diseases, comprising a skin-penetrating nucleic acid complex as an effective component, and more specifically, to a pharmaceutical composition or a cosmetic composition for preventing, alleviating, or treating atopic dermatitis, comprising a skin-penetrating nucleic acid complex in which a carrier peptide nucleic acid and a bioactive nucleic acid targeting TLR2 or IL-4Rα are connected by complementary binding. The skin-penetrating nucleic acid complex according to the present invention, in which the carrier peptide nucleic acid and the bioactive nucleic acid targeting TLR2 or IL-4Rα are connected by complementary binding, has high skin permeation and skin retention, and thus is useful in the prevention, alleviation or treatment of skin diseases such as atopic dermatitis.

This invention relates to long non-coding RNAs (lncRNAs), libraries of those lncRNAs that bind chromatin modifiers, such as Polycomb Repressive Complex 2, inhibitory nucleic acids and methods and compositions for targeting lncRNAs.

The invention is directed to one or more antisense polynucleotides and their use in pharmaceutical compositions in a strategy to induce exon skipping in the γ-sarcoglycan gene in patients suffering from Limb-Girdle Muscular Dystrophy-2C (LGM-D2C) or in patients at risk of such a disease. The invention also provides methods of preventing or treating muscular dystrophy, e.g., LGMD2C, by exon skipping in the gamma sarcoglycan gene using antisense polynucleotides. Accordingly, in some aspects the invention provides an isolated antisense oligonucleotide, wherein the oligonucleotide specifically hybridizes to an exon target region of a γ-sarcoglycan RNA. In another aspect, the the invention provides a method of inducing exon-skipping of a gamma sarcoglycan RNA, comprising delivering an antisense oligonucleotide or a composition to a cell.

The invention relates generally to viral infections and more specifically to compositions and methods for treating infections by SARS-CoV-2 (2019-nCoV). In particular, the invention provides a PNA agent which includes a PNA moiety with a sequence that targets a SARS-CoV-2 gene; a first cationic and hydrophobic peptide at the N-terminus of the PNA moiety, wherein the first peptide comprises lysine residues, and at least one of the lysine residues comprises a palmitoyl side chain moiety; and a second cationic and hydrophobic peptide at the C-terminus of the PNA moiety, wherein the second peptide comprises lysine residues, and at least one of the lysine residues comprises a palmitoyl side chain moiety.

Described herein is a modified anti-seed PNA including 5′-Xaa.sub.1Xaa.sub.2Xaa.sub.3-N.sub.1N.sub.2N.sub.3N.sub.4N.sub.5N.sub.6N.sub.7N.sub.8 N.sub.9-Xaa.sub.4-3′, wherein Xaa.sub.1, Xaa.sub.2, Xaa.sub.3, and Xaa.sub.4 are each independently R or K, wherein N.sub.1N.sub.2N.sub.3N.sub.4N.sub.5N.sub.6N.sub.7N.sub.8 N.sub.9 is a PNA which Watson-Crick base pairs to a seed sequence of an miRNA, wherein N.sub.8 and N.sub.9 may be null. Also included are methods of inhibiting expression of a miRNA in vivo or in vitro, and methods of treating cancer and other diseases and disorders with the modified anti-see PNAs.

The present disclosure provides compositions and methods related to nucleic acid molecules having therapeutic aptamers. In particular, the present disclosure provides nucleic acids molecules comprising one or more aptamers having anticoagulant activity, as well as corresponding nucleic acid antidotes, for the modulation of blood coagulation in the context of disease and surgical intervention.

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.

A method of reducing the level of a transcription product in a cell comprising contacting with the cell a composition comprising a double-stranded nucleic acid complex comprising a first nucleic acid strand annealed to a second nucleic acid strand, wherein: (i) the first nucleic acid strand hybridizes to the transcription product and comprises (a) a region consisting of at least 4 consecutive nucleotides that are recognized by RNase H when the strand is hybridized to the transcription product, (b) one or more nucleotide analogs located on 5′ terminal side of the region, (c) one or more nucleotide analogs located on 3′ terminal side of the region and (d) a total number of nucleotides and nucleotide analogs ranging from 8 to 35 nucleotides and (ii) the second nucleic acid strand comprises (a) nucleotides and optionally nucleotide analogs and (b) at least 4 consecutive RNA nucleotides.

Provided is an agent that binds to a wild type or variant pre-miRNA-1229 comprising a G-quadruplex (GQ) structure, wherein binding of the agent to the wild type or variant pre-miRNA-1229 stabilizes the GQ structure of the wild type or variant pre-miRNA-1229. In some embodiments, the variant is rs2291418. Provided is a method of treating a disease in a subject, comprising: administering a therapeutically effective amount of the agent to the subject. In some embodiments, the disease is Alzheimer's disease, cancer or coronary artery calcification.