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Novel adeno-associated viral vectors for retinal gene therapy

Gene Therapy volume 19, pages 162–168 (2012)Cite this article

An Erratum to this article was published on 24 November 2011

Abstract

Vectors derived from adeno-associated virus (AAV) are currently the most promising vehicles for therapeutic gene delivery to the retina. Recently, subretinal administration of AAV2 has been demonstrated to be safe and effective in patients with a rare form of inherited childhood blindness, suggesting that AAV-mediated retinal gene therapy may be successfully extended to other blinding conditions. This is further supported by the great versatility of AAV as a vector platform as there are a large number of AAV variants and many of these have unique transduction characteristics useful for targeting different cell types in the retina including glia, epithelium and many types of neurons. Naturally occurring, rationally designed or in vitro evolved AAV vectors are currently being utilized to transduce several different cell types in the retina and to treat a variety of animal models of retinal disease. The continuous and creative development of AAV vectors provides opportunities to overcome existing challenges in retinal gene therapy such as efficient transfer of genes exceeding AAV's cargo capacity, or the targeting of specific cells within the retina or transduction of photoreceptors following routinely used intravitreal injections. Such developments should ultimately advance the treatment of a wide range of blinding retinal conditions.

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Acknowledgements

We thank Dr Therese Cronin and Dr Robin Ali for the discussion and critical reading of the manuscript. Supported in part by: Grant Number UL1RR024134 (LHV) from the National Center for Research Resources and Institute for Translational Medicine and Therapeutics’ (ITMAT) Transdisciplinary Program in Translational Medicine and Therapeutics; the Foundation Fighting Blindness (LHV and AA), the European Commission under the FP7 AAVEYE project (Grant No HEALTH-2007-B-223445) (AA); the FP7 TREATRUSH project (Grant No 242013) (AA).

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  1. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA

    L H Vandenberghe

  2. Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy

    A Auricchio

  3. Department of Pediatrics, Medical Genetics, Federico II University, Naples, Italy

    A Auricchio

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  1. L H Vandenberghe
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Correspondence to L H Vandenberghe or A Auricchio.

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LHV is co-inventor of AAV technologies including novel serotypes which are described in patents licensed to various pharmaceutical and biotechnological companies.

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Vandenberghe, L., Auricchio, A. Novel adeno-associated viral vectors for retinal gene therapy. Gene Ther 19, 162–168 (2012). https://doi.org/10.1038/gt.2011.151

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