Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rhoâ/â mouse

As gene therapies for various forms of retinal degeneration progress toward human clinical trial, it will be essential to have a repertoire of safe and efficient vectors for gene delivery to the target cells. Recombinant adeno-associated virus (AAV) serotype 2/2 has been shown to be well tolerated i...

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Bibliographic Details
Main Authors: Arpad Palfi, Naomi Chadderton, Mary O'Reilly, Kerstin Nagel-Wolfrum, Uwe Wolfrum, Jean Bennett, Peter Humphries, Paul Kenna, Sophia Millington-Ward, Jane Farrar
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116300286