The present disclosure provides methods of treating subjects having glaucoma or elevated intraocular pressure (IOP), methods of identifying subjects having an increased risk of developing glaucoma or developing elevated IOP, methods of detecting human Rho Guanine Nucleotide Exchange Factor 12 (ARHGEF12) variant nucleic acid molecules and variant polypeptides, and ARHGEF12 variant nucleic acid molecules and variant polypeptides.

Provided are compositions related to HSD17B13 variants, including isolated nucleic acids and proteins related to variants of HSD17B13, and cells comprising those nucleic acids and proteins. Also provided are methods related to HSD17B13 variants. Such methods include methods for modifying a cell through use of any combination of nuclease agents, exogenous donor sequences, transcriptional activators, transcriptional repressors, and expression vectors for expressing a recombinant HSD17B13 gene or a nucleic acid encoding an HSD17B13 protein. Also provided are therapeutic and prophylactic methods for treating a subject having or at risk of developing chronic liver disease.

Provided herein are gene editing systems and/or compositions comprising RNA guides targeting LDHA for use in genetic editing of the LDHA gene. Also provide herein are methods of using the gene editing system for introducing edits to the LDHA gene and/or for treatment of primary hyperoxaluria (PH), and processes for characterizing the gene editing system.

The present invention relates to peptides, proteins, nucleic acids and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer. The present invention furthermore relates to tumor-associated T-cell peptide epitopes, alone or in combination with other tumor-associated peptides that can for example serve as active pharmaceutical ingredients of vaccine compositions that stimulate anti-tumor immune responses, or to stimulate T cells ex vivo and transfer into patients. Peptides bound to molecules of the major histocompatibility complex (MHC), or peptides as such, can also be targets of antibodies, soluble T-cell receptors, and other binding molecules.

A double stranded RNA interference (RNAi) agent comprising at least one of (i) a first double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a CD320 gene wherein the first dsRNA comprises a sense strand and an antisense strand forming a duplex, (ii) a second dsRNA for inhibiting the expression of a LRP2 gene wherein the second dsRNA comprises a sense strand and an antisense strand forming a duplex, or (iii) a cocktail of (i) and (ii) and wherein the sense strand of the first dsRNA is at least substantially complementary to the antisense strand of the first dsRNA and the sense strand of the second dsRNA is at least substantially complementary to the antisense strand of the second dsRNA and the use of the RNAi agent as a pharmaceutical composition for the treatment of cancer in subjects in need of treatment.

The present invention relates to ligand conjugates of oligonucleotides (e.g., iRNA agents) and methods for their preparation. The ligands are derived primarily from monosaccharides These conjugates are useful for the in vivo delivery of oligonucleotides.

The invention relates to methods of treating fibrosis and inflammatory bowel disease. In one embodiment, the present invention treats gut inflammation by administering a therapeutically effective dosage of TL1A inhibitors and/or DR3 inhibitors to an individual. In another embodiment, the present invention provides a method of reversing tissue fibrosis in an individual by inhibiting TL1A-DR3 signaling function.

The present invention pertains to a pharmaceutical composition for preventing or treating squamous cell carcinoma, the pharmaceutical composition containing a highly expressed lncRNAs in esophageal squamous cell carcinoma (HERES) expression inhibitor. More specifically, the present invention pertains to a pharmaceutical composition which uses a HERES expression inhibitor to reduce the expression of HERES and affect Wnt signaling pathways, and thereby prevent or treat squamous cell carcinoma.

The present inventors discovered that the expression pattern of HERES is related to the onset of squamous cell carcinoma, and found that HERES can be a target for treating squamous cell carcinoma. Accordingly, the pharmaceutical composition according to the present invention was determined to have the effect of inhibiting the proliferation, metastasis, and the like of squamous cell carcinoma by containing the HERES expression inhibitor, and is thus expected to be advantageously used for preventing and treating or ameliorating squamous cell carcinoma.

The present discloses relates to a composition, a kit or a method using an eIF4E inhibitor for diagnosis or treatment of a condition associated with increased activity of eIF4E.

Disclosed is use of a reagent for down-regulating the circular RNA-0007535 expression in preparation of drugs for preventing and/or treating pulmonary fibrosis and drugs for down-regulating the circular RNA-0007535 expression, belonging to the technical field of pharmaceutical preparation. The present disclosure provides a use of a reagent for down-regulating the circular RNA-0007535 expression in preparation of drugs for preventing and/or treating pulmonary fibrosis, the nucleotide sequence of the circular RNA-0007535 is set forth in SEQ ID NO. 1. Drugs prepared from the reagent for down-regulating the circular RNA-0007535 expression may prevent and/or treat pulmonary fibrosis, the cyclic RNA-0007535, as a potential molecular and drug target for treating pulmonary fibrosis diseases, provides a new perspective and field for exploring a regulation mechanism of gene expression in the occurrence and development of pulmonary fibrosis and searching for an intervention/drug, and has good application prospects.