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Review
. 2017 Jan 6;120(1):33-35.
doi: 10.1161/CIRCRESAHA.116.310039.

Introducing Genes to the Heart: All About Delivery

Roger J Hajjar  1 , Kiyotake Ishikawa  2
Affiliations
Review

Introducing Genes to the Heart: All About Delivery

Roger J Hajjar et al. Circ Res. .

Abstract

Recent clinical gene therapy trials for the treatment of heart failure have failed to meet primary efficacy endpoints and have tampered enthusiasm for the future application of cardiac gene therapy. These results have brought to light the difficulty of efficiently introducing genes into the human heart and have focused on potential problems that need to be addressed before further clinical applications. These trials however have established the safety of gene delivery vectors for cardiac targeting in humans. The sinusoidal trajectory of gene therapy continues and despite these setbacks the future of the field is promising.

Keywords: adeno-associated vectors; gene therapy; heart failure; sarcoplasmic reticulum calcium ATPase.

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References

    1. Greenberg B, Butler J, Felker GM, Ponikowski P, Voors AA, Desai AS, et al. Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial. Lancet. 2016;387:1178–1186. - PubMed
    1. Chung ES, Miller L, Patel AN, Anderson RD, Mendelsohn FO, Traverse J, et al. Changes in ventricular remodelling and clinical status during the year following a single administration of stromal cell-derived factor-1 non-viral gene therapy in chronic ischaemic heart failure patients: the STOP-HF randomized Phase II trial. Eur Heart J. 2015;36:2228–2238. - PMC - PubMed
    1. Hammond H, Penny WF, Traverse JH, et al. Intracoronary gene transfer of adenylyl cyclase 6 in patients with heart failure: A randomized clinical trial. JAMA Cardiology. 2016 - PMC - PubMed
    1. Hulot JS, Ishikawa K, Hajjar RJ. Gene therapy for the treatment of heart failure: promise postponed. Eur Heart J. 2016 - PMC - PubMed
    1. Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab. 2003;80:148–158. - PubMed

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