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

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

Mutations in the Crumbs homologue 1 (<i>CRB1</i>) 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 <i>Crb1<...

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Bibliographic Details
Main Authors: Nanda Boon, C. Henrique Alves, Aat A. Mulder, Charlotte A. Andriessen, Thilo M. Buck, Peter M. J. Quinn, Rogier M. Vos, Abraham J. Koster, Carolina R. Jost, Jan Wijnholds
Format: Article
Language:English
Published: MDPI AG 2021-03-01
Series:International Journal of Molecular Sciences
Subjects:
CRB1
CRB2
Crumbs homologue 1
retinitis pigmentosa
AAV tropism
AAV-mediated gene therapy
Online Access:https://www.mdpi.com/1422-0067/22/7/3563

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