vectors with enhanced photoreceptor targeting for retinal gene therapy. Localization and distribution of Müller glia cells (CRALBP, red) in retinal organoids. Nuclear DAPI staining (blue).
US FDA grants Orphan Drug Designation to AAVantgarde Bio’s AAVB-081 to treat Usher Syndrome Type 1B retinitis pigmentosa: Milan, Italy Tuesday, December 3, 2024, 16:00 Hrs [IST] ...
Francesca Simonelli Session title: Multicentric longitudinal study in Usher Syndrome IB: disease course and implication for gene therapy Session: Inherited retinal diseases treatments About the ...