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Defining Phenotype, Tropism, and Retinal Gene Therapy Using Adeno-Associated Viral Vectors (AAVs) in New-Born Brown Norway Rats with a Spontaneous Mutation in Crb1

Mutations in the Crumbs homologue 1 (CRB1) gene cause inherited retinal dystrophies, such as early-onset retinitis pigmentosa and Leber congenital amaurosis. A Brown Norway rat strain was reported with a spontaneous insertion-deletion (indel) mutation in exon 6 of Crb1. It has been reported that the...

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Detalles Bibliográficos
Autores principales: Boon, Nanda, Alves, C. Henrique, Mulder, Aat A., Andriessen, Charlotte A., Buck, Thilo M., Quinn, Peter M. J., Vos, Rogier M., Koster, Abraham J., Jost, Carolina R., Wijnholds, Jan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8036486/
https://www.ncbi.nlm.nih.gov/pubmed/33808129
http://dx.doi.org/10.3390/ijms22073563