Unfolding the therapeutic potential of chemical chaperones for age-related macular degeneration

Abstract

Recent studies suggest that pathological processes involved in age-related macular degeneration (AMD) might induce endoplasmic reticular (ER) stress. Growing evidence demonstrates the ability of chemical chaperones to decrease ER stress and ameliorate ER stress-related disease phenotypes, suggesting that the field of chaperone therapy might hold novel treatments for AMD. In this review, we examine the evidence suggesting a role for ER stress in AMD. Furthermore, we discuss the use of chaperone therapy for the treatment of ER stress-associated diseases, including other neurodegenerative diseases and retinopathies. Finally, we examine strategies for identifying potential chaperone compounds and for experimentally demonstrating chaperone activity in in vitro and in vivo models of human disease.

Keywords::

• AMD
• chemical chaperone
• endoplasmic reticulum stress
• pharmacological chaperone
• protein misfolding
• therapy

Financial & competing interests disclosure

Supported by the NEI Intramural Research Program, the Howard Hughes Medical Institute, and American Health Assistance Foundation. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.