Topical Administration of Novel FKBP12 Ligand MP-004 Improves Retinal Function and Structure in Retinitis Pigmentosa Models.

ABSTRACT:

Purpose

This study evaluates the therapeutic potential of MP-004, a novel FKBP12 ligand, in the treatment of inherited retinal dystrophies (IRDs). MP-004 targets the FKBP12/RyR interaction, which is disrupted in several neurologic disorders with underlying oxidative stress.

Methods

The toxicity and efficacy of MP-004 were examined in vitro in 661W cells. Efficacy was evaluated in phototoxic and H2O2-induced damage using impedance assays, calcium imaging, and in situ PLA. In vivo, MP-004 efficacy was evaluated in the rd10 mouse model of retinitis pigmentosa (RP) by topical ocular instillation. Retinal function was assessed by electroretinography (ERG), visual acuity was measured using a water maze test, and retinal structure was analyzed morphometrically.

Results

MP-00

SUBMITTER: Lara-Lopez A 

PROVIDER: S-EPMC11951062 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature