434. Long-Term Effects on Retinal Function and Structure Using AAV-Mediated Gene Therapy in Blind RPE65 Null Mutation Dogs

Kristina Narfström ,Greg Tullis ,Ragnheidur Bragadottir ,Mathias W Seeliger ,Elizabeth Rakoczy ,Martin L Katz

doi:10.1016/j.ymthe.2005.06.436

434. Long-Term Effects on Retinal Function and Structure Using AAV-Mediated Gene Therapy in Blind RPE65 Null Mutation Dogs

Kristina Narfström , Greg Tullis + Show 4 more

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https://doi.org/10.1016/j.ymthe.2005.06.436

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Journal: Molecular Therapy	Publication Date: May 1, 2005
License type: cc-by-nc-nd

#Human Leber's Congenital Amaurosis #Congenital Amaurosis + Show 8 more

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Abstract

We have previously reported on successful gene transfer using recombinant adeno-associated virus-mediated (rAAV) subretinal gene delivery for the dog model of human Leber's Congenital Amaurosis. Congenitally blind dogs were treated at 4-30 months of age with similar doses of the rAAV. RPE65 construct into the right eye.

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