Probing the sub-cellular mechanisms of LCA5-Leber Congenital Amaurosis and associated gene therapy with expansion microscopy
Siebren Faber, Olivier Mercey, Katrin Junger, Alejandro Garanto, Marius Ueffing, Rob W.J. Collin, Karsten Boldt, Paul Guichard, Virginie Hamel, Ronald Roepman
This study uses expansion microscopy to investigate LCA5-related photoreceptor defects and evaluates gene therapy efficacy in a mouse model.
bioRxiv · Jan 17, 2023 · PreprintMolecular Biology