Pharmacological modulation of the VCP/ERAD/proteasome axis rescues photoreceptor degeneration in P23H rhodopsin

Purpose One of the most prevalent mutations in rhodopsin (Rh) causing autosomal dominant retinitis pigmentosa (adRP) Rh P23H , results in its misfolding and its retention in the Endoplasmic Reticulum (ER). VCP is an ATP driven molecular checkpoint at the ER before these proteins are released from the ER towards plasma membrane. Misfolded proteins are selectively identified, retained at the ER and cleared by a process called ER‐associated...