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Prog Retin Eye Res. 2014 Nov;43:108-28. doi: 10.1016/j.preteyeres.2014.08.001. Epub 2014 Aug 12.

Vector platforms for gene therapy of inherited retinopathies.

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Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy.
Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy; Medical Genetics, Department of Translational Medicine, Federico II University, Naples, Italy. Electronic address:


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 either on rational design or directed evolution have generated vector variants with improved transduction properties. Dozens of promising proofs of concept have been obtained in IR animal models with both viral and non-viral vectors, and some of them have been relayed to clinical trials. To date, recombinant vectors based on the adeno-associated virus (AAV) represent the most promising tool for retinal gene therapy, given their ability to efficiently deliver therapeutic genes to both PR and RPE and their excellent safety and efficacy profiles in humans. However, AAVs' limited cargo capacity has prevented application of the viral vector to treatments requiring transfer of genes with a coding sequence larger than 5 kb. Vectors with larger capacity, i.e. nanoparticles, adenoviral and lentiviral vectors are being exploited for gene transfer to the retina in animal models and, more recently, in humans. This review focuses on the available platforms for retinal gene therapy to fight inherited blindness, highlights their main strengths and examines the efforts to overcome some of their limitations.


Adeno-associated virus; Adenovirus; Gene therapy; Inherited retinopathies; Lentivirus; Non-viral vectors

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