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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Detalhes bibliográficos
Publicado no:Front Neurosci
Principais autores: Palfi, Arpad, Yesmambetov, Adlet, Millington-Ward, Sophia, Shortall, Ciara, Humphries, Pete, Kenna, Paul F., Chadderton, Naomi, Farrar, G. Jane
Formato: Artigo
Idioma:Inglês
Publicado em: Frontiers Media S.A. 2020
Assuntos:
Neuroscience
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7482550/
https://ncbi.nlm.nih.gov/pubmed/32973439
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fnins.2020.00891
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