Article

Fine-tuning FAM161A gene augmentation therapy to restore retinal function

2023-10-06

Abstract excerpt

<h4>ABSTRACT</h4> In 15 years, inherited retinal diseases have seen gene therapy as a springboard to hope. Many preclinical investigations focused on vectors with maximal gene expression capabilities. But despite an efficient gene transfer, little physiological improvement was noted for certain ciliopathies. FAM161A is an essential protein for the structure of photoreceptor connecting cilium (CC). In the absence...

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Literature Corpus work: 9eb1e3d3-1d0d-5ac3-97de-7145e249aebf
DOI: 10.1101/2023.10.06.561164

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