Effective clinical delivery of gene therapy to the heart requires understanding and design of complex biological systems to deliver therapeutic gene expression. The development of vectors that specifically target the myocardium, in particular bioengineered recombinant viruses, has improved the efficiency of gene delivery to the heart. These tools, coupled with advances in selection and design of the genetic payload, have led to effective cardiac gene therapy in preclinical models. This technology is currently translating to the clinic with a new wave of gene therapy trials for myocardial disease.
- adeno-associated virus
- gene therapy
- genetic vectors
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Competing interests: RH is the scientific founder of Celladon Inc. and Nanocor, two biotechnology companies who are commercialising AAV gene therapy for heart failure. JS is a founder of Nanocor and Asklepios, start up companies focused on using AAV vectors for gene transfer. The other authors have no conflicts of interest to declare.