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Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models.

Hum. Mol. Genet.. 2015; 
Pellissier Lucie P,Quinn Peter M,Alves C Henrique,Vos Rogier M,Klooster Jan,Flannery John G,Heimel J Alexander,Wijnholds
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Custom Vector Construction Promoters, cDNA and poly-adenylation sequence were synthe- sized at Genscript and subcloned in pAAV vectors containing ITRs via Bgl II restriction sites. Get A Quote

摘要

Mutations in the Crumbs-homologue-1 (CRB1) gene lead to severe recessive inherited retinal dystrophies. Gene transfer therapy is the most promising cure for retinal dystrophies and has primarily been applied for recessive null conditions via a viral gene expression vector transferring a cDNA encoding an enzyme or channel protein, and targeting expression to one cell type. Therapy for the human CRB1 disease will be more complex, as CRB1 is a structural and signaling transmembrane protein present in three cell classes: Müller glia, cone and rod photoreceptors. In this study, we applied CRB1 and CRB2 gene therapy vectors in Crb1-retinitis pigmentosa mouse models at mid-stage disease. We tested if CRB expr... More

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