Successful gene therapy in older Rpe65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65.

@article{Annear2013SuccessfulGT,
  title={Successful gene therapy in older Rpe65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65.},
  author={Matthew J. Annear and Freya M Mowat and Joshua T. Bartoe and Janice Querubin and S A Sultana Azam and Mark Basche and Paul G. Curran and Alexander D. Smith and James W B Bainbridge and Robin R Ali and Simon Michael Petersen-Jones},
  journal={Human gene therapy},
  year={2013},
  volume={24 10},
  pages={883-93}
}
Young Rpe65-deficient dogs have been used as a model for human RPE65 Leber congenital amaurosis (RPE65-LCA) in proof-of-concept trials of recombinant adeno-associated virus (rAAV) gene therapy. However, there are relatively few reports of the outcome of rAAV gene therapy in Rpe65-deficient dogs older than 2 years of age. The purpose of this study was to investigate the success of this therapy in older Rpe65-deficient dogs. Thirteen eyes were treated in dogs between 2 and 6 years old. An rAAV2… CONTINUE READING