Patent classifications
C12N2310/113
MICRORNA-33 INHIBITORS AND USE THEREOF IN THE TREATMENT OF PULMONARY FIBROSIS
In one aspect, the present disclosure provides a microRNA-33 (miR-33) inhibitor comprising a peptide nucleic acid covalently bound to a pH low insertion peptide. In another aspect, the present disclosure provides a method of treating pulmonary fibrosis in a subject, the method comprising administering to the subject a therapeutically effective amount of the miR-33 inhibitor. In some embodiments, the pulmonary fibrosis is idiopathic pulmonary fibrosis.
MiRNA molecule, equivalent, antagomir, or source thereof for treating and/or diagnosing a condition and/or a disease associated with neuronal deficiency or for neuronal (re)generation
The invention relates to the diagnostic and therapeutic uses of a miRNA molecule, an equivalent or a source thereof in a neuronal deficiency or a disease and condition associated with neuronal deficiency.
Inhibitors of micro-RNA 22
The present disclosure provides compositions and methods that inhibit the activity of microRNAs, for example miR-22.
TARGETING MICRORNA FOR CANCER TREATMENT
This invention is directed to, inter alia, compositions and methods for restoring normal microRNA (miR) expression in chondrosarcoma cells as well as methods for treating and diagnosing chondrosarcoma in individuals in need thereof.
3′-UTR sequences for stabilization of RNA
The present invention relates to stabilization of RNA, in particular mRNA, and an increase in mRNA translation. The present invention particularly relates to a modification of RNA, in particular in vitro-transcribed RNA, resulting in increased transcript stability and/or translation efficiency. According to the invention, it was demonstrated that certain sequences in the 3′-untranslated region (UTR) of an RNA molecule improve stability and translation efficiency.
ANTISENSE OLIGONUCLEOTIDES FOR TREATMENT OF CANCER STEM CELLS
The invention provides oligonucleotides complementary to a non-coding chimeric mitochondrial RNA as well as compositions and kits comprising the same, and their use in treating and preventing metastasis or relapse of a cancer in an individual previously treated for cancer with a therapy. The invention also provides oligonucleotides complementary to a non-coding chimeric mitochondrial RNA as well as compositions and kits comprising the same, and their use in treating a refractory cancer (e.g., a refractory HPV-associated cancer).
COMPOSITIONS COMPRISING REVERSIBLY MODIFIED OLIGONUCLEOTIDES AND USES THEREOF
Disclosed herein are glutathione-sensitive oligonucleotides and methods of using the same. Any oligonucleotide of interest may be modified with a glutathione-sensitive moiety, including oligonucleotides used for in vivo delivery, such as nucleic acid inhibitor molecules. Typically, the glutathione-sensitive moiety is used to reversibly modify the 2′-carbon of a sugar moiety in one or more nucleotides in the oligonucleotide, although other carbon positions may also be modified with the glutathione-sensitive moiety. Also disclosed are glutathione-sensitive nucleotide and nucleoside monomers, including glutathione-sensitive nucleoside phosphoramidites that can be used, for example, in standard oligonucleotide synthesis methods. In addition, glutathione-sensitive nucleotide and nucleoside monomers without a phosphoramidite can be used therapeutically, for example, as anti-viral agents.
INCREASING GENE EXPRESSION
The present disclosure relates to compositions and methods for modulating gene expression and in particular to compositions and methods for increasing expression of Klotho. In certain examples, the present disclosure provides a method of increasing expression of a Klotho gene in a cell the method comprising administering to the cell a binding molecule that binds to an RNA transcript transcribed from a chromosomal region within or near the Klotho gene, wherein the RNA transcript does not encode a Klotho protein.
USE OF MIRNA-485 INHIBITORS FOR TREATING TAUOPATHY
The present disclosure includes the use of a miRNA inhibitor for treating a tauopathy associated with a decreased level of SIRT1 protein or SIRT1 gene expression, PGC-1α protein and/or PGC-α gene expression, and/or CD36 and/or CD36 gene expression.
PHARMACEUTICAL COMPOSITION CONTAINING HERES EXPRESSION INHIBITOR FOR PREVENTING OR TREATING SQUAMOUS CELL CARCINOMA
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.