Remedy for CAG repeat expansion diseases
First Claim
1. A method of treating a CAG repeat expansion disease comprising administering a therapeutically effective amount of a pharmaceutical composition comprising a transglutaminase inhibitor, wherein the transglutaminase inhibitor is selected from a group consisting of cystamine and monodanysl cadaverine.
3 Assignments
0 Petitions
Accused Products
Abstract
To elucidate the molecular mechanisms of “gain of toxic function” of expanded polyglutamine stretches in CAG repeat expansion diseases, the inventors established an expression system of full-length and truncated cDNAs for dentatorubral-pallidoluysian atrophy (DRPLA) and found that truncated DRPLA proteins containing the expanded polyglutamine stretch, but not the full-length protein, form peri- and intra-nuclear aggregates consisting of filaments and concomitant apoptosis. The apoptotic cell death was partially suppressed by transglutaminase inhibitors, cystamine and monodansyl cadaverine, raising the possibility of involvement of transglutaminase reaction. The results may provide a potential basis for the development of therapeutic measures for CAG repeat expansion diseases.
46 Citations
3 Claims
- 1. A method of treating a CAG repeat expansion disease comprising administering a therapeutically effective amount of a pharmaceutical composition comprising a transglutaminase inhibitor, wherein the transglutaminase inhibitor is selected from a group consisting of cystamine and monodanysl cadaverine.
-
2. A method of treating a CAG repeat expansion disease comprising administering a therapeutically effective amount of a pharmaceutical composition for treating a CAG repeat expansion disease, said pharmaceutical composition comprising a transglutaminase inhibitor as its ingredient, and at least one pharmaceutically accepted ingredient for formulation.
Specification