Ocular delivery of different VCP inhibitory formulations prevents retinal degeneration in rhodopsin 255 isoleucine deletion mice
Bowen Cao, Regine L. Mühlfriedel, Merve Sen, Ana-Cristina Almansa-Garcia, Mathias W. Seeliger, Anne-Sophie Petremann-Dumé, Ellen Kilger, Anneli Vollert, Sylvia Bolz, Christine Henes, Paolo Caliceti, Stefano Salmaso, Marius Ueffing, Blanca Arango‐González
This study evaluates three VCP-targeted therapies in a mouse model of retinitis pigmentosa, finding that nanocarrier-encapsulated ML240 provides the best protection against photoreceptor degeneration.
bioRxiv · Jun 25, 2025 · PreprintNeuroscience