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

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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Bibliographic Details
Main Authors: Scott H. Greenwald, Emily E. Brown, Michael J. Scandura, Erin Hennessey, Raymond Farmer, Basil S. Pawlyk, Ru Xiao, Luk H. Vandenberghe, Eric A. Pierce
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
NMNAT1
Leber congenital amaurosis
LCA9
gene therapy
AAV
NAD+
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301522

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http://www.sciencedirect.com/science/article/pii/S2329050120301522

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