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Review
. 2018 Aug 17;123(5):601-613.
doi: 10.1161/CIRCRESAHA.118.311587.

Human Cardiac Gene Therapy

Kiyotake Ishikawa  1 , Thomas Weber  1 , Roger J Hajjar  1
Affiliations
Review

Human Cardiac Gene Therapy

Kiyotake Ishikawa et al. Circ Res. .

Abstract

In the past 10 years, there has been tremendous progress made in the field of gene therapy. Effective treatments of Leber congenital amaurosis, hemophilia, and spinal muscular atrophy have been largely based on the efficiency and safety of adeno-associated vectors. Myocardial gene therapy has been tested in patients with heart failure using adeno-associated vectors with no safety concerns but lacking clinical improvements. Cardiac gene therapy is adapting to the new developments in vectors, delivery systems, targets, and clinical end points and is poised for success in the near future.

Keywords: AAV; Clinical trial; Leber congenital amaurosis; gene therapy; heart failure; myocardium.

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Figures

Figure 1:
Figure 1:
Timeline of gene therapy milestones over the last century
Figure 2:
Figure 2:
Factors that increase vector uptake in the myocardium.
Figure 3:
Figure 3:
A. Shuffling of AAV capsid genes to create mosaics. B. Selection of AAV vector variants that have high tropism to the heart.
See this image and copyright information in PMC

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