Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse modelsCRB2 in immature photoreceptors determines the superior-inferior symmetry of the developing retina to maintain retinal structure and functionProduction of iPS-Derived Human Retinal Organoids for Use in Transgene Expression AssaysThe CRB1 Complex: Following the Trail of Crumbs to a Feasible Gene Therapy StrategyHuman iPSC-Derived Retinas Recapitulate the Fetal CRB1 CRB2 Complex Formation and Demonstrate that Photoreceptors and Müller Glia Are Targets of AAV5Loss of CRB2 in Müller glial cells modifies a CRB1-associated retinitis pigmentosa phenotype into a Leber congenital amaurosis phenotype
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Peter M Quinn