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Compounds as modulators of a mutant CFTR protein and their use for treating diseases associated with cftr protein malfunction

A compound and mixture technology, applied in the field of novel protein regulators, can solve problems such as exocrine pancreatic duct obstruction

Inactive Publication Date: 2015-04-01
INST BIOCHEM I BIOFIZYKI POLSKIEJ AKADI NAUK
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Moreover, dysregulation of the CFTR protein leads to obstruction of the exocrine pancreatic duct and associated digestive disorders (13)

Method used

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  • Compounds as modulators of a mutant CFTR protein and their use for treating diseases associated with cftr protein malfunction
  • Compounds as modulators of a mutant CFTR protein and their use for treating diseases associated with cftr protein malfunction
  • Compounds as modulators of a mutant CFTR protein and their use for treating diseases associated with cftr protein malfunction

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Experimental program
Comparison scheme
Effect test

Embodiment

[0102] Materials and methods

[0103] Antibody

[0104] The following antibodies were used: MAB25031 (clone 24-1, R&D systems, USA) and MM13-4 (Upstate), monoclonal antibodies (mAb) for CFTR detection; fluorescent secondary antibodies Alexa 594 and 488 from Molecular Probes (Cergy Pontoise, France) purchase.

[0105] cell culture

[0106] Stably transfected HeLa cells (HeLa cells only contain the pTracer plasmid as a control (pTracer), or express WT-CFTR (spTCF-WT), ΔF508-CFTR (spTCF-F508del)) were prepared by Pascale Fanen (Inserm U.468, Créteil , France) and cultured as described elsewhere (16). Briefly, HeLa cells were cultured in Dulbecco's Modified Eagle Medium (DMEM) supplemented with 10% heat-inactivated FCS, 100 U / ml penicillin, 100 μg / ml streptomycin, and 250 μg / ml zeocin antibiotics. at 37°C in 5% CO 2 cultured in a humidified incubator. Expression of WT-CFTR and ΔF508-CFTR in these cells was verified by immunoprecipitation and immunocytochemistry throug...

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Abstract

The present invention relates to novel protein modulators capable of altering function of the mutant CFTR protein and their use for treating diseases associated with CFTR protein malfunction. The invention provides compositions, pharmaceutical preparations and methods of correcting the cellular alteration of a mutant CFTR protein wherein the CFTR mutation is a mutation delta F508-CFTR, or another mutation of class II.

Description

[0001] This application is an invention patent application submitted on June 20, 2011 with the title of "Compound as a Regulator of Mutant CFTR Protein and Its Use in the Treatment of Diseases Related to CFTR Protein Abnormality" and the application number "201180048679.6" divisional application. technical field [0002] The present invention relates to novel protein regulators capable of altering the function of mutant CFTR proteins, and their use in the treatment of diseases associated with CFTR protein malfunction. The present invention provides compositions, pharmaceutical formulations and methods for correcting cellular changes in CFTR mutant proteins, wherein the CFTR mutation is the mutation ΔF508-CFTR, or another class II mutation. Background technique [0003] Cystic fibrosis (also known as CF or mucoviscous disease) is one of the most common fatal genetic diseases in humans. CF is an inherited autosomal recessive disorder affecting approximately 1 in 2,500 live bi...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C07D473/22A61K31/52A61P43/00
CPCC07D219/10C07D473/22C07C233/65C07D473/30C07F9/305C07C2603/18A61K31/496A61K31/663A61K31/52A61K31/194C07D401/12A61P1/00A61P1/18A61P11/00A61P13/00A61P15/00A61P19/04A61P43/00A61P9/00C07F9/30A61K31/435C07C235/84C07F9/304
Inventor N. 奥多拉兹奇P. 杰伦凯维奇G. 维乔雷克A. 埃德尔曼D. 通德莱尔J. 弗里奇
Owner INST BIOCHEM I BIOFIZYKI POLSKIEJ AKADI NAUK
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