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Hum Gene Ther. 2017 Aug 16;:

Authors: Ylä-Herttuala S, Lähteenvuo J

25 years of gene therapy have not yet yielded standard therapeutic solutions for clinical use in cardiovascular medicine, but several therapeutic targets have been identified and foundations for future therapies have been set. The safety of viral gene therapy has been established with a wide variety of vectors and transgenes. Adenoviruses and adeno-associated viruses have established their role as vectors of choice for many cardiovascular applications and appropriate viral doses have been established for several tissues and applications. Local delivery methods are favored as opposed to systemic delivery. The first patients received gene therapy already over 20 years ago, and long term safety analyses have been performed. Further improvement of the method and success in clinical setting requires still more careful considerations of preclinical disease models, study settings and end points both in preclinical and clinical settings. We should not expect to see more in patients than we've seen in controlled laboratory settings, but often these studies have very different viewpoints, expectations and even methods. The gap between preclinical and clinical trials should be bridged from both ends. Animal models and preclinical study settings should be designed to better mimic the clinical target population and treatment goals, and clinical disappointments could be better utilized by more careful analysis of responders and non-responders. The reasons for unsuccessful clinical trials often remain speculative. Factors associated with poor outcomes in clinical trials could be reproduced in experimental setting and the contribution of individual factors could be more carefully analyzed. This review focuses on current status of cardiovascular gene therapy with emphasis on myocardial gene therapy targets and trials. We emphasize the value of bidirectional translationality from bench to bedside and back, and suggest novel approaches to better fulfill the clinical demand for novel therapies.

PMID: 28810808 [PubMed - as supplied by publisher]