Methods in Molecular Biology Volume 935, 2013, pp 351-369

Adeno-Associated Viral Vectors for Gene Therapy of Inherited Retinal Degenerations

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Abstract

Adeno-associated virus (AAV) vectors are in wide use for in vivo gene transfer for the treatment of inherited retinal disease. AAV vectors have been tested in many animal models and have demonstrated efficacy with low toxicity. In this chapter we describe some of the recent methods for small-scale production of these vectors for use in a laboratory setting in volumes and purity appropriate for testing in small and large animals.