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Genes 2017, 8(2), 51;

Viral Vector-Mediated Antisense Therapy for Genetic Diseases

INSERM U1179, Université de Versailles St-Quentin en Yvelines and Université Paris Saclay, 2 Avenue de la Source de la Bièvre, 78180 Montigny-le-Bretonneux, France
These authors contributed equally to this work.
Author to whom correspondence should be addressed.
Academic Editor: Selvarangan Ponnazhagan
Received: 28 October 2016 / Revised: 4 January 2017 / Accepted: 17 January 2017 / Published: 26 January 2017
(This article belongs to the Section Human Genomics and Genetic Diseases)
Full-Text   |   PDF [709 KB, uploaded 26 January 2017]   |  


RNA plays complex roles in normal health and disease and is becoming an important target for therapeutic intervention; accordingly, therapeutic strategies that modulate RNA function have gained great interest over the past decade. Antisense oligonucleotides (AOs) are perhaps the most promising strategy to modulate RNA expression through a variety of post binding events such as gene silencing through degradative or non-degradative mechanisms, or splicing modulation which has recently demonstrated promising results. However, AO technology still faces issues like poor cellular-uptake, low efficacy in target tissues and relatively rapid clearance from the circulation which means repeated injections are essential to complete therapeutic efficacy. To overcome these limitations, viral vectors encoding small nuclear RNAs have been engineered to shuttle antisense sequences into cells, allowing appropriate subcellular localization with pre-mRNAs and permanent correction. In this review, we outline the different strategies for antisense therapy mediated by viral vectors and provide examples of each approach. We also address the advantages and limitations of viral vector use, with an emphasis on their clinical application. View Full-Text
Keywords: antisense therapy; snRNA; viral vectors; splice-switching approaches antisense therapy; snRNA; viral vectors; splice-switching approaches

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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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Imbert, M.; Dias-Florencio, G.; Goyenvalle, A. Viral Vector-Mediated Antisense Therapy for Genetic Diseases. Genes 2017, 8, 51.

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