Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa

Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa

Despite mutations in the rod phosphodiesterase 6-alpha (PDE6A) gene being well-recognized as a cause of human retinitis pigmentosa, no definitive treatments have been developed to treat this blinding disease. We performed a trial of retinal gene augmentation in the Pde6a mutant dog using Pde6a deliv...

Full description

Bibliographic Details
Main Authors: Freya M. Mowat, Laurence M. Occelli, Joshua T. Bartoe, Kristen J. Gervais, Ashlee R. Bruewer, Janice Querubin, Astra Dinculescu, Sanford L. Boye, William W. Hauswirth, Simon M. Petersen-Jones
Format: Article
Language:English
Published: Frontiers Media S.A. 2017-06-01
Series:Frontiers in Neuroscience
Subjects:
retinitis pigmentosa
Pde6 mutation
gene therapy
canine model
adeno-associated virus
Online Access:http://journal.frontiersin.org/article/10.3389/fnins.2017.00342/full

Internet

http://journal.frontiersin.org/article/10.3389/fnins.2017.00342/full

Similar Items