AAV8-Mediated Gene Therapy Rescues Retinal Degeneration Phenotype in a Tlcd3b Knockout Mouse Model

PURPOSE: The purpose of this study was to assess the therapeutic efficacy of rAAV8-hGRK1-Tlcd3b in a Tlcd3b(−)(/)(−) mouse model of retinal generation and validate TLCD3B's role as a ceramide synthase in vivo. METHODS: Using Tlcd3b(−)(/)(−) mice as an inherited retinal disease animal model, we...

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Detalles Bibliográficos
Autores principales: Qian, Xinye, Liu, Hehe, Fu, Shangyi, Lu, Jiaxiong, Hung, Yu-Ting, Turner, Cassidy, Gu, Haiwei, Chen, Rui
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Association for Research in Vision and Ophthalmology 2022
Materias:
Retina
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8934561/
https://www.ncbi.nlm.nih.gov/pubmed/35275174
http://dx.doi.org/10.1167/iovs.63.3.11

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8934561/
https://www.ncbi.nlm.nih.gov/pubmed/35275174
http://dx.doi.org/10.1167/iovs.63.3.11

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