Presentation

Recovery of Cone-Mediated Vision in Lebercilin-Associated Severe Retinal Ciliopathy (LCA5) after Gene Therapy: One Year Results of a Phase Ib/IIa Trial

Relative preservation of the extramacular retina in LCA5-associated Leber congenital amaurosis

Treatment Potential for LCA5-Associated Leber Congenital Amaurosis

Uyhazi et al., Investigative Ophthalmology and Visual Science 2020

Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness

Song et al., Molecular Therapy 2018