Improved ocular gene transfer with a Neddylation-site modified AAV-RPE65 vector in rd12 mice
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DOI: 10.1038/s41433-020-0838-8
Abstract: Leber congenital amaurosis type 2 (LCA2) is an inherited disease that affects the integrity of the retina and results in severe visual impairment early in life [1]. This disease is caused due to mutations in… read more here.
Keywords: vector; aav2 vectors; ocular gene; mice ... See more keywords