Inactivation of cellular retinol-binding protein 1 protects against bis-retinoid accumulation and light-induced retinal degeneration in mice.

ABSTRACT: Delayed clearance of all-trans-retinal (at-RAL) in photoreceptors is linked to prevalent retinal diseases such as Stargardt disease, rod-cone dystrophies, and age-related macular degeneration. Pharmacological modulation of retinoid metabolism in the eye presents a promising therapeutic strategy, with cellular retinol-binding protein 1 (RBP1) emerging as a potential target. However, it lacks genetic validation as a therapeutic target in an animal model of human disease. Thus, we investigated the effect of the Rbp1 gene inactivation on the phenotype of the Abca4^-/-/Rdh8^-/- model of Stargardt disease. Triple knockout (Abca4^-/-/Rdh8^-/-/Rbp1^-/-) mice were protected against light-induced retinal degeneration. RBP1 deficiency also slowed bis-

SUBMITTER: Widjaja-Adhi MAK 

PROVIDER: S-EPMC12398791 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature