Article

Expression of NMNAT1 in the Photoreceptors is Sufficient to Prevent <i>NMNAT1</i> -Associated Disease

2022-11-21

Abstract excerpt

Nicotinamide nucleotide adenylyltransferase 1 (NMNAT1) is a ubiquitously expressed enzyme involved in nuclear NAD + production throughout the body. However, mutations in the NMNAT1 gene lead to retina-specific disease with few reports of systemic effects. We have previously demonstrated that AAV-mediated gene therapy using self-complimentary AAV (scAAV) to ubiquitously express NMNAT1 throughout the retina preven...

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Literature Corpus work: 0bf83426-d918-5fd6-b73e-fb32265d4f60
DOI: 10.1101/2022.11.20.517250

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