C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) G01N 33/50 (2006.01) G01N 33/68 (2006.01)
Patent
CA 2736425
The present invention relates to agents, and methods for identifying compounds, which agents and compounds result in the modulation of cellular trafficking of proteins in particular that of CF- associated mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). In addition, the invention relates to compositions and methods for the use thereof in treating conditions that are characterized by an ER-associated protein misfolding and abnormal cellular trafficking of disease-associated proteins, including cystic fibrosis (CF).
La présente invention porte sur des agents et sur des procédés permettant didentifier des composés, lesquels agents et composés produisant la modulation du trafic cellulaire de protéines, en particulier de celui du régulateur de conductance transmembranaire de la mucoviscidose mutant (CFTR) associé à la mucoviscidose (MV). De plus, l'invention porte sur des compositions et des procédés pour l'utilisation de celles-ci dans le traitement d'affections qui sont caractérisées par un mauvais repliement de protéines associées au RE et un trafic cellulaire anormal de protéines associées à une maladie, dont la mucoviscidose (MV).
Fischer David Frederik
Janssen Richard Antonius Jozef
Roseboom Marjet
Scaffidi Amelia Katie
Tessari Michela
Galapagos Nv
Norton Rose Or S.e.n.c.r.l. S.r.l./llp
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