Barriers in Heart Failure Gene Therapy and Approaches to Overcome Them

With the growing prevalence and incidence of heart failure worldwide, investigation and development of new therapies to address disease burden are of great urgency. Gene therapy is one promising approach for the management of heart failure, but several barriers currently exclude safe and efficient g...

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Bibliographic Details
Published in:Heart, lung & circulation lung & circulation, 2023-07, Vol.32 (7), p.780-789
Main Authors: Ravichandran, Anjali J., Romeo, Francisco J., Mazurek, Renata, Ishikawa, Kiyotake
Format: Article
Language:English
Subjects:
Adeno-associated virus
Adenoviridae - genetics
Adenovirus
Cardiomyocyte specificity
Gene Transfer Techniques
Genetic Therapy
Genetic Vectors
Heart failure
Heart Failure - genetics
Heart Failure - therapy
Humans
Immunogenicity
Transduction efficiency
Transduction, Genetic
Vector
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Summary:With the growing prevalence and incidence of heart failure worldwide, investigation and development of new therapies to address disease burden are of great urgency. Gene therapy is one promising approach for the management of heart failure, but several barriers currently exclude safe and efficient gene delivery to the human heart. These barriers include the anatomical and biological difficulty of specifically targeting cardiomyocytes, the vascular endothelium, and immunogenicity against administered vectors and the transgene. We review approaches taken to overcome these barriers with a focus on vector modification, evasion of immune responses, and heart-targeted delivery techniques. While various modifications proposed to date show promise in managing some barriers, continued investigation into improvements to existing therapies is required to address transduction efficiency, duration of transgene expression, and immune response.
ISSN:1443-9506
1444-2892
1444-2892
DOI:10.1016/j.hlc.2023.02.011