Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

Mutations in the cilia-centrosomal protein CEP290 are frequently observed in autosomal recessive childhood blindness disorder Leber congenital amaurosis (LCA). No treatment or cure currently exists for this disorder. The Cep290(rd16) (retinal degeneration 16) mouse (a model of LCA) carries a mutatio...

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Detalles Bibliográficos
Autores principales: Zhang, Wei, Li, Linjing, Su, Qin, Gao, Guangping, Khanna, Hemant
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc. 2018
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5770090/
https://www.ncbi.nlm.nih.gov/pubmed/28679290
http://dx.doi.org/10.1089/hum.2017.049

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5770090/
https://www.ncbi.nlm.nih.gov/pubmed/28679290
http://dx.doi.org/10.1089/hum.2017.049

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