Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion.

Adeno-associated virus (AAV) continues to be the gold standard vector for therapeutic gene delivery and has proven especially useful for treating ocular disease. Intravitreal injection (IVtI) is a promising delivery route because it increases accessibility of gene therapies to larger patient populations. However, data from clinical and non-human primate (NHP) studies utilizing currently available capsids indicate that anatomical barriers to AAV and pre-existing neutralizing antibodies can restrict gene expression to levels that are "sub-therapeutic" in a substantial proportion of patients.

Lifecycles of Individual Subretinal Drusenoid Deposits and Evolution of Outer Retinal Atrophy in Age-Related Macular Degeneration.

Purpose: To describe the progression and regression of individual subretinal drusenoid deposits (SDDs) and surrounding photoreceptors and retina in patients with age-related macular degeneration (AMD) over a 3.5-year period using multimodal imaging including adaptive optics scanning laser ophthalmoscopy (AOSLO).

Design: Longitudinal observational study.