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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Detalles Bibliográficos
Autores principales: Palfi, Arpad, Yesmambetov, Adlet, Millington-Ward, Sophia, Shortall, Ciara, Humphries, Pete, Kenna, Paul F., Chadderton, Naomi, Farrar, G. Jane
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7482550/
https://www.ncbi.nlm.nih.gov/pubmed/32973439
http://dx.doi.org/10.3389/fnins.2020.00891

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7482550/
https://www.ncbi.nlm.nih.gov/pubmed/32973439
http://dx.doi.org/10.3389/fnins.2020.00891

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