Neural Regeneration Research ›› 2022, Vol. 17 ›› Issue (1): 110-112.doi: 10.4103/1673-5374.314306

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

Yibo Xi, Yuanyuan Chen*   

  1. Department of Ophthalmology, University of Pittsburgh, Pittsburgh, PA, USA (Xi Y)
    Department of Pharmacology and Chemical Biology, University of Pittsburgh, Pittsburgh, PA, USA (Chen Y) 
    McGowan Institute of Regenerative Medicine, University of Pittsburgh, Pittsburgh, PA, USA (Chen Y)
  • Online:2022-01-05 Published:2021-09-18
  • Contact: Yuanyuan Chen, PhD, cheny1@pitt.edu.
  • Supported by:
    The present work was supported by the NIH Grants R01 EY030991 to YC and the P30 EY008098 to the Department of Ophthalmology at University of Pittsburgh, the Ear and Eye Foundation of Pittsburgh and an unrestricted grant from Research to Prevent Blindness, New York, NY, USA.

Abstract: 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. This perspective uses RHODOPSIN (RHO)-associated RP to review pharmacologic strategies for modifying protein misfolding-associated abnormalities with the goal of bringing insights to the treatment of other proteinopathies.