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Genes 2019, 10(4), 287; https://doi.org/10.3390/genes10040287

Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina

1
Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy
2
Medical Genetics, Department of Translational Medicine, Federico II University, 80131 Naples, Italy
Received: 1 March 2019 / Revised: 26 March 2019 / Accepted: 5 April 2019 / Published: 9 April 2019
(This article belongs to the Special Issue Molecular Therapies for Inherited Retinal Diseases)
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Abstract

Gene therapy using adeno-associated viral (AAV) vectors currently represents the most promising approach for the treatment of many inherited retinal diseases (IRDs), given AAV’s ability to efficiently deliver therapeutic genes to both photoreceptors and retinal pigment epithelium, and their excellent safety and efficacy profiles in humans. However, one of the main obstacles to widespread AAV application is their limited packaging capacity, which precludes their use from the treatment of IRDs which are caused by mutations in genes whose coding sequence exceeds 5 kb. Therefore, in recent years, considerable effort has been made to identify strategies to increase the transfer capacity of AAV vectors. This review will discuss these new developed strategies, highlighting the advancements as well as the limitations that the field has still to overcome to finally expand the applicability of AAV vectors to IRDs due to mutations in large genes. View Full-Text
Keywords: AAV; retina; gene therapy; dual AAV AAV; retina; gene therapy; dual AAV
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Trapani, I. Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina. Genes 2019, 10, 287.

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