Article

A drug-tunable gene therapy for broad-spectrum protection against retinal degeneration

2018-03-16

Abstract excerpt

Retinal degenerations are a large cluster of diseases characterized by the irreversible loss of light-sensitive photoreceptors that impairs the vision of 9.1 million people in the US. An attractive treatment option is to use gene therapy to deliver broad-spectrum neuroprotective factors. However, this approach has had limited clinical translation because of the inability to control transgene expression. To address...

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Literature Corpus work: 513e2ffe-6a1d-5599-a0aa-69935809c84f
DOI: 10.1101/283887

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