Vector platforms for gene therapy of inherited retinopathies.

  title={Vector platforms for gene therapy of inherited retinopathies.},
  author={Ivana Trapani and Agostina Puppo and Alberto Auricchio},
  journal={Progress in retinal and eye research},
Inherited retinopathies (IR) are common untreatable blinding conditions. Most of them are inherited as monogenic disorders, due to mutations in genes expressed in retinal photoreceptors (PR) and in retinal pigment epithelium (RPE). The retina's compatibility with gene transfer has made transduction of different retinal cell layers in small and large animal models via viral and non-viral vectors possible. The ongoing identification of novel viruses as well as modifications of existing ones based… CONTINUE READING
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