37 results about "Polycystic liver disease" patented technology

The present invention relates to the polycystic kidney disease 1 (PKD1) gene and its nucleic acid sequence, mutations thereof in patients having PKD1-associated disorders, the protein encoded by the PKD1 gene or its mutants, and their uses in disease diagnosis and therapy.

The present invention is directed to methods of treating or ameliorating an effect of a polycystic disease. More particularly, the methods include administering to a patient in need thereof an amount of a modulator of a tumor necrosis factor (TNF) pathway, which is sufficient to treat or ameliorate an effect of a polycystic disease. Methods of treating or ameliorating an effect of a polycystic kidney disease (PKD) are also provided. Methods are also provided for identifying a candidate compound that may be effective to treat or ameliorate an effect of a polycystic disease or to increase polycystin-2 (PC2) function or decrease Rab11-Family of Interacting Protein2 (FIP2) function. Further provided are methods for identifying a patient having, or who is at risk for developing, a polycystic disease or who would benefit from treatment with a TNF-alpha inhibitor.

An object of the present invention is to provide a combination drug that has remarkably excellent preventive and / or therapeutic effects on polycystic kidney disease. The present invention provides a drug for preventing and / or treating polycystic kidney disease comprising a combination of tolvaptan or a prodrug thereof with a somatostatin derivative, and a method for treating polycystic kidney disease using this drug.

The present invention provides compounds of Formula (I) or (II), which are thought to be able to inhibit mTOR (mammalian target of rapamycin) signaling pathway, induce UPR (unfolded protein response), and / or perturb mitochondrial function of a cyst cell (e.g., a cyst cell causing polycystic kidney disease (PKD, e.g., autosomal dominant PKD (ADPKD) or autosomal recessive PKD (ARPKD)) or polycystic liver disease (PLD, e.g., autosomal dominant PLD (ADPLD) or autosomal recessive PLD (ARPLD)). The invention also provides pharmaceutical compositions, kits, and methods involving the compounds described herein for use in treating PKD or PLD, inhibiting the growth of a cyst cell, and / or killing a cyst cell.

Disclosed is a method of treating or preventing polycystic kidney disease (PKD) by administration of neutrophil geleatinase-associated lipocalin (Ngal) protein. Also, a transgenic non-human animal model is established to investigate the effect of overexpression of exogenous Ngal on PKD progression.

This invention describes the use of indane acetic acid derivatives which are dual PPAR delta / gamma agonists for the treatment of liver diseases including one or more of the following: NAFLD (Non Alcoholic Fatty Liver Disease), NASH (Non Alcoholic Steatohepatitis), Farber's Disease, ACLF (Acute-on-Chronic Liver Failure), CLF (Chronic Liver Failure), POLT-HCV-SVR (Post-Orthotopic Liver Transplantdue to Hepatitis C Virus infection after Sustained Viral Response following anti-HCV therapy), Alagille syndrome, PFIC (Progressive Familial Intrahepatic Cholestasis), PBC (Primary Biliary Cirrhosis),Primary Sclerosing Cholangitis, ADPCLD (Autosomal Dominant Polycystic Liver Disease), Treatment of liver transplant patients with reestablished fibrosis, CESD (Cholesteryl Ester Storage Disease), SHTG (Severe Hypertriglyceridemia), HoFH (Homozygous Familial Hypercholesterolemia), HE (Hepatic Encephalopathy), or Alcoholic Liver Disease.

In order to determine the epigenetic mutation of autosomal dominant polycystic kidney disease and its functional relevance, the present inventors randomly analyzed the whole genome of polycystic kidney disease and non-polycystic kidney disease individuals by methylation profiling method and compared with their performance data. Interestingly, PKD1 and other genes associated with ion transport and cell binding were hypermethylated at the gene body, which was downregulated in polycystic kidney disease. In particular, PKD1 is partially hypermethylated in the polycystic kidney gene body, which is associated with MBD2 (methyl‑CpG‑binding domain 2) protein binding. Furthermore, DNA methylation inhibitor treatment was accompanied by an upregulation of PKD1 expression and delayed cyst formation in MDCK (Madin‑Darby Canine Kidney) cells. Thus, hypermethylation of PKD1 and cyst formation-associated regulatory genes of the present invention is decisive for cyst formation and revealed to be useful as a treatment for autosomal dominant polycystic kidney disease.