Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration

No treatment is available for nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1)-associated retinal degeneration, an inherited disease that leads to severe vision loss early in life. Although the causative gene, NMNAT1, plays an essential role in nuclear nicotinamide adenine dinucleotide (NA...

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Veröffentlicht in:Mol Ther Methods Clin Dev
Hauptverfasser: Greenwald, Scott H., Brown, Emily E., Scandura, Michael J., Hennessey, Erin, Farmer, Raymond, Pawlyk, Basil S., Xiao, Ru, Vandenberghe, Luk H., Pierce, Eric A.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2020
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7397406/
https://ncbi.nlm.nih.gov/pubmed/32775493
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.07.003
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