use of a compound selected from the group consisting of a proteasome inhibitor, an autophagy inhibitor, a lysosomal inhibitor, an golgi er protein transport inhibitor, an hsp90 chaperonin inhibitor, a heat shock response activator, a and a histone deacetylase inhibitor, use of 11-cis-retinal or 9-cis-retinal and a compound selected from the group consisting of a proteasome inhibitor, an autophagy inhibitor, a lissosomal inhibitor, a er to golgi protein, a hsp90 chaperonin inhibitor, a heat shock response activator, a glycosidase inhibitor, and a histone deacetylase inhibitor, a method for increasing the amount of a biochemically functional conformation of a protein in a cell, composition pharmaceutical for the treatment of an ocular pcd, pharmaceutical composition for the treatment of retinitis pigmentosa, kit for the treatment of an ocular pcd, kit for the treatment retinitis pigmentosa, method for identifying a compound useful for the treatment of an individual having an ocular pcd,

摘要

USE OF A SELECTED COMPOUND OF THE GROUP THAT CONSISTS OF A PROTEASOMAL INHIBITOR, AN AUTOPHAGY INHIBITOR, A LISOSOMAL INHIBITOR, AN INHIBITOR OF TRANSPORTATION OF ER TO GOLGI, AN INHIBITOR OF CHAPERONINE HSP9O, AN ACTIVATOR OF CHAPERONINE, AN ACTIVATOR OF GLYCOSIDASE, AND A HISTONE DEACETYLASE INHIBITOR, USE OF 11-CIS-RETINAL OR 9-CIS-RETINAL AND A SELECTED COMPOSITION OF THE GROUP THAT CONSISTS OF A PROTEASOMAL INHIBITOR, AN INHIBITOR INHIBITOR, A LISSOSOMAL INHIBITOR INHIBITOR PROTEIN FROM ER TO GOLGI, A CHAPERONIN INHIBITOR HSP9O, AN ACTIVATOR OF RESPONSE TO THERMAL SHOCK, A GLYCOSIDASE INHIBITOR, AND A DEACETYLASE HISTONE INHIBITOR, A METHOD OF INCREASING THE QUANTITY OF A BIOCHEMICAL CONTAINMENT PHARMACEUTICAL FOR THE TREATMENT OF AN EYE PCD, PHARMACEUTICAL COMPOSITION FOR THE TREATMENT OF PIGMENTAL RETINITIS, KIT FOR THE TREATMENT OF AN EYE PCD, KIT FOR THE TREATMENT OF PIGMENTAL RETINITIS, METHOD FOR THE IDENTIFICATION OF A USEFUL COMPOUND FOR THE TREATMENT OF AN INDIVIDUAL THAT HAS AN EYE PCD, METHOD FOR THE IDENTIFICATION OF A USEFUL COMPOUND FOR TREATING AN INDIVIDUAL THAT HAS PIGMENTAL RETINITIS, AN INHIBITOROUS DISEASE OR INHIBITOROUS DISEASE, OR INHIBITOROUS DISEASE. AUTOPHAGY AND METHOD FOR THE PRODUCTION OF A RECOMBINANT PROTEIN IN A BIOCHEMICALLY FUNCTIONAL CONFORMATION The invention features compositions and methods that are useful for the treatment or prevention of a protein-forming disease in an individual by correcting proteins in vivo. In addition, the invention features compositions and methods that are useful for expressing a recombinant protein in a biochemically functional conformation.