Conductance of improperly folded proteins through the secretory pathway and related methods for treating disease
OwnerMichael J. Caplan, Marie E. Eganinventors
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Abstract
This invention provides the methodology and agents for treating any 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.
Claims36
A method of preventing, treating, or alleviating symptoms of any disease or clinical condition, which condition is at least partly the result of endoplasmic reticulum-associated retention or degradation of mis-assembled or mis-folded proteins, the method comprising steps of:
- identifying an individual at risk of or suffering from a condition that is at least partly the result of endoplasmic reticulum-associated retention or degradation of mis-assembled or mis-folded proteins;
- and
- administering a composition comprising curcumin, a curcumin related compound, or an analog or derivative of curcumin to the individual so that the disease or clinical condition is treated, prevented, or its symptoms relieved.
A composition comprising an siRNA targeted to a transcript encoding a molecule selected from the group consising of:
- UDP glucose:glycoprotein glycosyl transferase, calnexin or endoplasmic reticulum Ca++ ATPase, or a chaperone involved in retention of a misfolded or misassembled protein in the endoplasmic reticulum.
A cell engineered or manipulated to contain an siRNA targeted to a transcript encoding a molecule selected from the group consising of:
- UDP glucose:glycoprotein glycosyl transferase, calnexin or endoplasmic reticulum Ca++ ATPase, or a chaperone involved in retention of a misfolded or misassembled protein in the endoplasmic reticulum.
A composition comprising a nebulized or aerosolized formulation of curcumin, a curcumin related compound, or an analog or derivative of curcumin.
Dependencies are inferred from the claim text.