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Cardiac gene therapy: are we there yet?

Gene Therapy volume 23, pages 635–648 (2016)Cite this article

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Abstract

The incidence of cardiovascular disease (CVD) is increasing throughout the world and is associated with elevated morbidity and mortality. Gene therapy to treat cardiac dysfunction is gaining importance because of the limited therapeutic benefit offered by pharmacotherapies. The growing knowledge of the complex signaling pathways and the development of sophisticated vectors and delivery systems, are facilitating identification and targeting of specific molecular candidates involved in initiation and progression of CVDs. Several preclinical and clinical studies have shown the therapeutic efficiency of gene therapy in different disease models and patients. Hence, gene therapy might plausibly become an unconventional treatment modality for CVD patients. In this review, we summarize the gene delivery carriers, modes of delivery, recent preclinical/clinical studies and potential therapeutic targets. We also briefly discuss the existing limitations of gene therapy, technical challenges surrounding gene carriers and delivery systems, and some approaches to overcome these limitations for bringing CVD gene therapy one step closer to reality.

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Acknowledgements

We thank members of the Leong-Poi lab and our collaborators at St Michael’s Hospital for their valuable contributions to some of the results discussed in this review.

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Authors and Affiliations

  1. Department of Cardiology at Keenan Research Centre for Biomedical Science, St. Michael's Hospital, Toronto, Ontario, Canada

    P N Matkar & H Leong-Poi

  2. Department of Vascular Surgery at Keenan Research Centre for Biomedical Science, St. Michael's Hospital, Toronto, Ontario, Canada

    P N Matkar, H Leong-Poi & K K Singh

  3. Department of Vascular Surgery, St Michael’s Hospital, Toronto, Ontario, Canada

    K K Singh

  4. Department of Cardiac Surgery at Keenan Research Centre for Biomedical Science, St. Michael’s Hospital, Toronto, Ontario, Canada

    K K Singh

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  1. P N Matkar
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Correspondence to K K Singh.

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Matkar, P., Leong-Poi, H. & Singh, K. Cardiac gene therapy: are we there yet?. Gene Ther 23, 635–648 (2016). https://doi.org/10.1038/gt.2016.43

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