A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/00 (2006.01) A61K 31/02 (2006.01) A61K 31/122 (2006.01) A61K 31/343 (2006.01) A61K 31/365 (2006.01) A61K 31/407 (2006.01) A61K 31/55 (2006.01) A61P 3/06 (2006.01) A61P 5/00 (2006.01) A61P 5/48 (2006.01) A61P 7/00 (2006.01) A61P 11/00 (2006.01) A61P 31/12 (2006.01) A61P 35/00 (2006.01) C12Q 1/48 (2006.01) G01N 33/48 (2006.01)
Patent
CA 2348529
This invention provides the methodology and agents for treating disease or clinical condition which is at least partly the result of endoplasmic reticulum-associated retention of proteins. Thus, the methods and agents of the present invention provide for the release of normally retained proteins from the endoplasmic reticulum. The present invention is particularly useful for treating any disease or clinical condition which is at least partly the result of endoplasmic reticulum-associated retention or degradation of mis- assembled or mis-folded proteins. In particular, thapsigargin, cyclopiazonic acid, DBHQ or halothane can be used to allow the release of .DELTA.F508CFTR or of a secretion-incompetent variant null (Hong Kong) of .alpha.1-antitrypsin, in order to treat cystic fibrosis or chronic obstructive pulmonary disease (pulmonary emphysema) respectively.
L'invention concerne une méthodologie et des agents permettant de traiter les maladies ou états cliniques dus, en partie au moins, à la rétention de protéines associée au réticulum endoplasmique. Ainsi, les techniques et les agents de la présente invention permettent la libération par le réticulum endoplasmique des protéines qu'il retient normalement. La présente invention est particulièrement utile pour traiter les maladies ou états cliniques résultant, en partie au moins, de la rétention ou de la dégradation associée au réticulum endoplasmique de protéines mal assemblées ou mal repliées.
Caplan Michael J.
Egan Marie E.
Robic
Yale University
LandOfFree
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