72 results about "POLYCYSTIC RENAL DISEASE" patented technology

The present invention provides a method for treating, inhibiting the progression of, or eradicating polycystic kidney disease of in a patient in need thereof which comprises providing to said patient an effective amount of a TACE inhibitor compound in combination with an effective amount of a Src kinase inhibitor, a HER-2 kinase inhibitor, or a combination of Src and HER-2 inhibitor.

Conformationally constrained compounds that mimic the secondary structure of reverse-turn regions of biologically active peptides and proteins are disclosed. Such reverse-turn mimetic structures have utility over a wide range of fields, including use as diagnostic and therapeutic agents. Libraries containing the reverse-turn mimetic structures of this invention are also disclosed as well as methods for screening the same to identify biologically active members. The invention also relates to the use of such compounds for inhibiting or treating disorders modulated by Wnt-signaling pathway, such as cancer, especially colorectal cancer, restenosis associated with angioplasty, polycystic kidney disease, aberrant angiogenesis disease, rheumatoid arthritis disease, tuberous sclerosis complex, Alzheimer's disease, excess hair growth or loss, or ulcerative colitis.

Conformationally constrained compounds that mimic the secondary structure of reverse-turn regions of biologically active peptides and proteins are disclosed. Such reverse-turn mimetic structures have utility over a wide range of fields, including use as diagnostic and therapeutic agents. Libraries containing the reverse-turn mimetic structures of this invention are also disclosed as well as methods for screening the same to identify biologically active members. The invention also relates to the use of such compounds for inhibiting or treating disorders modulated by Wnt-signaling pathway, such as cancer, especially colorectal cancer, restenosis associated with angioplasty, polycystic kidney disease, aberrant angiogenesis disease, rheumatoid arthritis disease, tuberous sclerosis complex, Alzheimer's disease, excess hair growth or loss, or ulcerative colitis.

This invention provides compounds of Formula (I), having the structurewhere T, Z, X, A, R<1>, R<2a>, R<2b>, R<2c>, R<3>, R<4>, and n are defined herein, or a pharmaceutically acceptable salt thereof which are useful as antineoplastic agents and in the treatment of osteoporosis and polycystic kidney disease.

Conformationally constrained compounds that mimic the secondary structure of reverse-turn regions of biologically active peptides and proteins are disclosed. Such reverse-turn mimetic structures have utility over a wide range of fields, including use as diagnostic and therapeutic agents. Libraries containing the reverse-turn mimetic structures of this invention are also disclosed as well as methods for screening the same to identify biologically active members. The invention also relates to the use of such compounds for inhibiting or treating disorders modulated by Wnt-signaling pathway, such as cancer, especially colorectal cancer, restenosis associated with angioplasty, polycystic kidney disease, aberrant angiogenesis disease, rheumatoid arthritis disease, tuberous sclerosis complex, Alzheimer's disease, excess hair growth or loss, or ulcerative colitis.

Conformationally constrained compounds that mimic the secondary structure of reverse-turn regions of biologically active peptides and proteins are disclosed. Such reverse-turn mimetic structures have utility over a wide range of fields, including use as diagnostic and therapeutic agents. Libraries containing the reverse-turn mimetic structures of this invention are also disclosed as well as methods for screening the same to identify biologically active members. The invention also relates to the use of such compounds for inhibiting or treating disorders modulated by Wnt-signaling pathway, such as cancer, especially colorectal cancer, restenosis associated with angioplasty, polycystic kidney disease, aberrant angiogenesis disease, rheumatoid arthritis disease, tuberous sclerosis complex, Alzheimer's disease, excess hair growth or loss, or ulcerative colitis.

Systems and methods are provided for locally delivering specific prophylactic, regenerative, or therapeutic agents within the body of a patient. Systems and methods can involve direct delivery of prophylactic, regenerative, or therapeutic agents into branch blood vessels or body lumens from a main vessel or lumen, respectively, and in particular into renal arteries extending from an aorta in a patient. Drug infusion techniques encompass specific treatment and prevention regimes for renal diseases including, but not limited to, Acute Kidney Injury, Renal Cell Carcinoma, Polycystic Kidney Disease, and Chronic Kidney Disease.

Methods and composition provided herein relate to the inhibition of proliferation, migration, and tubule formation of cells and are thus useful in treating angiogenesis associated diseases, including cancer, polycystic kidney disease, diabetic retinopathy, rheumatoid arthritis, and psoriasis. Disclosed are methods of inhibiting endothelial cell proliferation, migration, and tubule formation by administering an antibody specific for Tumor EndothPelial Markers (TEMs). Also disclosed are methods of inhibiting angiogenesis and tumor growth by administering a TEM-specific antibody and antibody compositions useful in such methods.

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.

Conformationally constrained compounds that mimic the secondary structure of reverse-turn regions of biologically active peptides and proteins are disclosed. Such reverse-turn mimetic structures have utility over a wide range of fields, including use as diagnostic and therapeutic agents. Libraries containing the reverse-turn mimetic structures of this invention are also disclosed as well as methods for screening the same to identify biologically active members. The invention also relates to the use of such compounds for inhibiting or treating disorders modulated by Wnt-signaling pathway, such as cancer, especially colorectal cancer, restenosis associated with angioplasty, polycystic kidney disease, aberrant angiogenesis disease, rheumatoid arthritis disease, tuberous sclerosis complex, Alzheimer's disease, excess hair growth or loss, or ulcerative colitis.

A method of treating polycystic kidney disease in a subject comprises administering to the subject an effective amount of a compound represented by Structural Formula (1): or a pharmaceutically acceptable salt thereof.

Provided herein are highly water soluble, thiazolidinone derivative compounds and glycine hydrazide derivative compounds that inhibit the ion transport activity of the cystic fibrosis transmembrane conductance regulator (CFTR). The compounds, and compositions comprising the compounds, described herein are useful for treating diseases, disorders, and sequelae of diseases, disorders, and conditions that are associated with aberrantly increased CFTR activity, for example, secretory diarrhea. The compounds may also be used for inhibiting expansion or preventing formation of cysts in persons who have polycystic kidney disease.

The present invention relates to a kind of pyrimidine substituted phenylpropionic acid derivative compounds of formula I which includes itsgeometric isomer, enantiomer, diastereoisomer, racemate, and mixture therefore or pharmaceutically acceptable salt thereof. The present invention also relates to the preparation method and use of such compound. Compounds of present invention are useful in the treatment of polycystic kidney disease.

Compositions and methods are provided for identifying agents which have efficacy for the treatment of disorders related to aberrant cilial structure and function, including polycystic kidney disease.

Compounds of formula (I) wherein: the groups are as defined in the description, are useful for the preparation of medicaments for the treatment of cardiovascular disorders, in particular heart failure and hypertension. The compounds are inhibitors of the enzymatic activity of the Na+,K+-ATPase. They are useful for the preparation of a medicament for the treatment of a disease caused by the hypertensive effects of endogenous ouabain, such as renal failure progression in autosomal dominant polycystic renal disease (ADPKD), preeclamptic hypertension and proteinuria and renal failure progression in patients with adducin polymorphisms.

A method of treating polycystic kidney disease in a subject comprises administering to the subject an effective amount of a compound represented by Structural Formula (1):or a pharmaceutically acceptable salt thereof.

The present invention relates to compositions and methods for treating a disease in an animal, which disease is responsive to inhibiting of functional cystic fibrosis transmembrane conductance regulator (CFTR) polypeptide by administering to a mammal in need thereof an effective amount of a compound defined herein (including those compounds set forth in Tables 1-3 or encompassed by formula I-III) or compositions thereof, thereby treating the disease. The present invention particularly, relates to a method of treating diarrhea and polycystic kidney disease.

Compositions and methods for supporting health, especially renal health, comprising ketonic agents that recapitulate beneficial effects of ketosis by exogenously administered agents. The agents include BHB, analogs thereof, and GPR109A agonists. The agents may further include crystal precipitation inhibitors which synergistically improve treatment of certain renal conditions. The agents may be used in dietary supplements and therapeutic compositions for the treatment of cystic kidney diseases such as polycystic kidney disease, ciliopathies, and other conditions.

The invention belongs to the technical field of gene knockout, and discloses a CRISPR Cas9 conditional gene knockout mouse and an establishment method. According to the establishment method, a vectoris designed and constructed, and gRNA and Cas9 protein are prepared; a gRNA sequence and a targeting vector are designed; the targeting vector is constructed by the In-Fusion technology, and the targeting vector is verified by restriction digestion, PCR and sequencing; the gRNA and Cas9 protein are prepared at the same time; microinjection and F0 mouse identification are performed; the obtained Donor vector, gRNA and Cas9 protein are co-injected into a fertilized egg; the fertilized egg after microinjection is returned to the oviduct of a surrogate mouse; PCR and sequencing identification areperformed after a mouse is born, and a positive F0 mouse is obtained; the reproduction and identification of a F1 generation mouse are performed; the sexually mature positive F0 mice are matched withwild-type mice respectively for reproducing the first generation, and a polycystic kidney gene PKD1 knockout mouse is obtained successfully.

The present invention relates to novel heteroaryl amide derivatives of formula (I), as selective inhibitors of histone deacetylase 1 and 2 (hdac1-2), to methods for the production of same, to pharmaceutical compositions comprising these compounds, and to the use of said compounds for the production of a medicament for the treatment of pathological conditions or diseases that can be improved by means of the inhibition of the activity of histone deacetylase class I, particularly HDAC1 and HDAC2, such as cancer, neurodegenerative diseases, infectious diseases, inflammatory diseases, heart failureand cardiac hypertrophy, diabetes, polycystic kidney disease, sickle cell disease and beta-thalassemia disease, and to methods for the treatment of the diseases mentioned above.

A new method of in vitro monitoring and assessing the need of a medication which interferes with the regulation of the parathyroid hormone level in a kidney patient subject to oxidative stress, notably hemodialysis patients. FIG. 1 shows the distribution of n-oxPTH concentrations in 340 hemodialysis patients (224 men and 116 women) with a median age of 66 years (IQR, 56 to 75 years), a median time since initiation of dialysis (dialysis vintage) of 266 days (IQR, 31 to 1209 days), and a median dialysis dose (kt / V) of 1.2 (IQR, 1.1 to 1.3). The cause of chronic kidney disease was nephrosclerosis in 113 cases (33%), diabetic nephropathy in 107 cases (31%), chronic glomerular nephritis in 29 cases (9%), polycystic kidney disease in 9 cases (3%) and other / unknown in 82 cases (24%). The median n-oxPTH concentration was 5.9 ng / L (IQR, 2.4 to 14.0 ng / L). n-oxPTH concentrations were not different in men and women (5.9 ng / L; IQR, 2.4 to 14.2 ng / L; n=224; vs. 5.5 ng / L; IQR, 2.4 to 14.0 ng / L; n=1 16; p=0.915).

Provided herein are pyrimido-pyrrolo-quinoxalinedione (PPQ) compounds, and compositions comprising these compounds, that inhibit cystic fibrosis transmembrane conductance regulator (CFTR) mediated ion transport and that are useful for treating diseases and disorders associated with aberrantly increased CFTR chloride channel activity. The compounds, and compositions comprising the compounds, described herein are useful for treating diseases, disorders, and sequelae of diseases, disorders, and conditions that are associated with aberrantly increased CFTR activity, for example, polycystic kidney disease. The compounds may be used for inhibiting expansion or preventing formation of cysts in persons who have polycystic kidney disease.

Compounds of formula (I) wherein: the groups are as defined in the description, are useful for the preparation of medicaments for the treatment of cardiovascular disorders, in particular heart failure and hypertension. The compounds are inhibitors of the enzymatic activity of the Na<+>,K<+>-ATPase. Said compounds are used for the preparation of a medicament for the treatment of a disease caused by the hypertensive effects of endogenous ouabain, such as renal failure progression in autosomal dominant polycystic renal disease (ADPKD), preeclamptic hypertension and proteinuria and renal failure progression in patients with adducin polymorphisms.

The present invention discloses new uses of a Shh signaling pathway inhibitor. The new uses of the Shh signaling pathway inhibitor specifically comprises applications of the Shh signaling pathway inhibitor in preparation of autosomal domi-nant polycystic kidney disease (ADPKD) treatment product, wherein the Shh signaling pathway inhibitor is specifically Vismodegib. According to the present invention, a Vil-Cre:Pkd2f3 / f3 mouse as an ADPKD animal model, and the results show that with the Shh signaling pathway inhibitor Vismodegib, the renal cyst degree of the Vil-Cre:Pkd2f3 / f3 mouse can be reduced, the kidney weight ration of the Vil-Cre:Pkd2f3 / f3 mouse can be reduced, the contents of creatinine and urea nitrogen in the blood of the Vil-Cre:Pkd2f3 / f3 mouse can be reduced, and the renal fibrosis degree of the Vil-Cre:Pkd2f3 / f3 mouse can be reduced; and the important significance is provided for the analysis of the ADPKD pathogenesis and the research and development of the ADPKD treatment drug.

The invention relates to a pharmaceutical composition for treating polycystic kidney disease. The active ingredients of the pharmaceutical composition comprise effective doses of thiazolidinediones and diuretics. The preferential combination is as follows: the thiazolidinediones are selected from rosiglitazone and the diuretics are selected from amiloride hydrochloride. The animal experiments prove that the efficacy of the composition of the rosiglitazone and the diuretics on the treatment of the polycystic kidney disease is better than the single use of one drug. The effect of the pharmaceutical composition for treating the polycystic kidney disease is better than the single use of one drug, thereby being a drug for treating the polycystic kidney disease and being worth expecting.

Compounds of structure formula (I) or a pharmaceutically salt thereof are useful as antineoplastic agents and in the treatment of osteoporosis and polycystic kidney disease.

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.

The reagent composition for detecting the autosomal dominant polycystic kidney disease comprises a primer group, a first reagent for extracting genomic DNA from a sample; the second reagent is used for carrying out long fragment PCR reaction by using the primer group; a third agent for processing the amplification product such that the amplification product can be used in a high-throughput sequencing technique; and the fourth reagent is used for performing high-throughput sequencing on the treated amplification product. Comprising the following steps: S1, amplifying a PKD1 gene by using a reagent composition according to any one of claims 1-7 through long-fragment PCR (Polymerase Chain Reaction); s2, performing high-throughput sequencing on the amplified sequence obtained in the step (1); s3, comparing the sequencing result obtained in the step (2) with a PKD1 gene reference sequence, and determining a mutation site; s4, eliminating false gene locus interference through bioinformatics analysis, and determining a PKD1 true gene mutation locus; and S5, comparing the true gene mutation site determined in the step S4 with a known PKD1 gene mutation site, and determining a new mutation site on the PKD1 gene.