Pharmacological strategies for treating misfolded rhodopsin- associated autosomal dominant retinitis pigmentosa

摘要

Mutations that cause protein misfolding are implicated in conditions such as retinitis pigmentosa(RP),Usher Syndrome,and myocilin associated primary open angle glaucoma.The aggregation and continuous degradation of a highly abundant misfolded protein add proteolytic load of the affected cells.The subtle balance of cellular homeostasis,once disrupted by an overwhelmed proteolytic system,will lead to cell death and tissue degeneration.