Looking for breakthrough ideas for innovation challenges? Try Patsnap Eureka!

Compounds as modulators of mutant cftr proteins and their use in the treatment of diseases associated with cftr protein disorders

A protein disorder, F508-CFTR technology, applied in the field of new protein regulators, can solve problems such as exocrine pancreatic duct obstruction

Inactive Publication Date: 2016-08-31
INST BIOCHEM I BIOFIZYKI POLSKIEJ AKADI NAUK
View PDF11 Cites 0 Cited by
  • 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

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
View more

Image

Smart Image Click on the blue labels to locate them in the text.
Viewing Examples
Smart Image
  • Compounds as modulators of mutant cftr proteins and their use in the treatment of diseases associated with cftr protein disorders
  • Compounds as modulators of mutant cftr proteins and their use in the treatment of diseases associated with cftr protein disorders
  • Compounds as modulators of mutant cftr proteins and their use in the treatment of diseases associated with cftr protein disorders

Examples

Experimental program
Comparison scheme
Effect test

Embodiment

[0100] Materials and methods

[0101] Antibody

[0102] 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 (CergyPontoise, France )Buy.

[0103] cell culture

[0104] 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 throughout th...

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to View More

PUM

No PUM Login to View More

Abstract

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 dysfunctional CFTR proteins. The present invention provides compositions, pharmaceutical formulations and methods for correcting cellular changes in mutant CFTR proteins, wherein the CFTR mutation is the mutation ΔF508-CFTR, or another Type II mutation.

Description

technical field [0001] 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 [0002] 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 births (1). CF is caused by mutations in the cftr gene, which encodes the cystic fibrosis transmembrane transport regulator (CFTR protein) with epithelial chloride channel activity (2,3). As a result of impaired function of this protein, severe symptoms related to the respiratory, digestive, and male...

Claims

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to View More

Application Information

Patent Timeline
no application Login to View More
Patent Type & Authority Patents(China)
IPC IPC(8): C07D219/10C07D473/30C07F9/30C07C233/65A61K31/663A61K31/435
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
Who we serve
  • R&D Engineer
  • R&D Manager
  • IP Professional
Why Patsnap Eureka
  • Industry Leading Data Capabilities
  • Powerful AI technology
  • Patent DNA Extraction
Social media
Patsnap Eureka Blog
Learn More
PatSnap group products

Browse by: Latest US Patents, China's latest patents, Technical Efficacy Thesaurus, Application Domain, Technology Topic, Popular Technical Reports.

© 2024 PatSnap. All rights reserved.Legal|Privacy policy|Modern Slavery Act Transparency Statement|Sitemap|About US| Contact US: help@patsnap.com