AAV-Delivered Tulp1 Supplementation Therapy Targeting Photoreceptors Provides Minimal Benefit in Tulp1−/− Retinas

AAV-Delivered Tulp1 Supplementation Therapy Targeting Photoreceptors Provides Minimal Benefit in Tulp1−/− Retinas

With marketing approval of the first ocular gene therapy, and other gene therapies in clinical trial, treatments for inherited retinal degenerations (IRDs) have become a reality. Biallelic mutations in the tubby like protein 1 gene (TULP1) are causative of IRDs in humans; a mouse knock-out model (Tu...

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Bibliographic Details
Main Authors: Arpad Palfi, Adlet Yesmambetov, Sophia Millington-Ward, Ciara Shortall, Pete Humphries, Paul F. Kenna, Naomi Chadderton, G. Jane Farrar
Format: Article
Language:English
Published: Frontiers Media S.A. 2020-08-01
Series:Frontiers in Neuroscience
Subjects:
retina
eye
mouse
disease model
inherited retinal degeneration
Tulp1
Online Access:https://www.frontiersin.org/article/10.3389/fnins.2020.00891/full

Internet

https://www.frontiersin.org/article/10.3389/fnins.2020.00891/full

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