Archives: Presentations
Presentations
RDH12 Mutations Cause a Severe Retinal Degeneration With Relatively Spared Rod Function
Aleman et al., Investigative Ophthalmology and Visual Science 2018
Mutation-Independent Rhodopsin Gene Therapy by Knockdown and Replacement with a Single AAV Vector
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness
Song et al., Molecular Therapy 2018
BEST1 Gene Therapy Corrects a Diffuse Retina-Wide Microdetachment Modulated by Light Exposure
Bestrophinopathy: An RPE-Photoreceptor Interface Disease
Mouse Models of NMNAT1-Leber Congenital Amaurosis (LCA9) Recapitulate Key Features of the Human Disease
Greenwald et al., American Journal of Pathology 2016
Gene Augmentation for AdRP Mutations in RHO
Recombinant AAV-Mediated BEST1 Transfer to the Retinal Pigment Epithelium: Analysis of Serotype-Dependent Retinal Effects
NMNAT1 mutations cause Leber congenital amaurosis.
Falk et al., Nature Genetics 2012
