Article

Targeting ON-bipolar cells by AAV gene therapy stably reverses <i>LRIT3</i> -congenital stationary night blindness

2021-10-04

Abstract excerpt

AAV gene therapies aimed at curing inherited retinal diseases to date have typically focused on photoreceptors and retinal pigmented epithelia within the relatively accessible outer retina. However, therapeutic targeting in diseases such as congenital stationary night blindness (CSNB) that involve defects in ON-bipolar cells (ON-BCs) within the mid-retina has been challenged by the relative inaccessibility of the...

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Literature Corpus work: 7db90660-1267-53a9-b807-a0b16ded0733
DOI: 10.1101/2021.10.04.462588

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