Article
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.
International journal of molecular sciences - 30 Mar 2021
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
Abstract excerpt
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 these Crb1 mutant rats show vascular abnormalities associated with retinal telangiectasia and possess an early-onset...
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