Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration-Reference-Cited by-同舟云学术

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

Published:2020-09 Issue: Volume:18 Page:582-594
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Greenwald Scott H.,Brown Emily E.,Scandura Michael J.,Hennessey Erin,Farmer Raymond,Pawlyk Basil S.,Xiao Ru,Vandenberghe Luk H.,Pierce Eric A.

Funder

Foundation Fighting Blindness

National Eye Institute

Sara Elizabeth O'Brien Trust

Fight for Sight

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference76 articles.

1. Exome sequencing identifies NMNAT1 mutations as a cause of Leber congenital amaurosis;Chiang;Nat. Genet.,2012

2. Mutations in NMNAT1 cause Leber congenital amaurosis and identify a new disease pathway for retinal degeneration;Koenekoop;Nat. Genet.,2012

3. Mutations in NMNAT1 cause Leber congenital amaurosis with early-onset severe macular and optic atrophy;Perrault;Nat. Genet.,2012

4. NMNAT1 mutations cause Leber congenital amaurosis;Falk;Nat. Genet.,2012

5. NMNAT1 variants cause cone and cone-rod dystrophy;Nash;Eur. J. Hum. Genet.,2018

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2. Inhibitors of NAD+ Production in Cancer Treatment: State of the Art and Perspectives;International Journal of Molecular Sciences;2024-02-08

3. Gene augmentation therapy attenuates retinal degeneration in a knockout mouse model of Fam161a retinitis pigmentosa;Molecular Therapy;2023-10

4. NAD+ metabolism and eye diseases: current status and future directions;Molecular Biology Reports;2023-08-04

5. The landscape of basic gene therapy approaches in inherited retinal dystrophies;Frontiers in Ophthalmology;2023-06-27

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