| 摘要 |
The use of a farnesyl transferase inhibitor, FTI-277 to enhance autophagic protein degradation for the treatment or prevention of a protein conformation disorder (PCD) selected from the group consisting of alpha1-antitrypsin deficiency, Huntington's disease, nephrogenic diabetes insipidus, Jacob-Creutzfeld disease, glaucoma (excluding neo-vascular glaucoma), corneal dystrophies, retinoschises, Stragardt's disease autosomal dominant druzen and Best's macular dystrophy. The treatment can further comprise the use of 11-cis-retinal, 9-cis-retinal or a 7-ring locked isomer of 11-cis-retinal for treating an ocular PCD. In particular the farnesyl transferase inhibitor or FTI-277 inhibits the protein mammalian target of rapamycin (mTOR) or the protein Ras homolog enriched in brain (Rheb).
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