title: Current Methods in Cardiac Gene Therapy: Overview.
abstract: During the last decade, there has been a significant progress toward clinical translation in the field of cardiac gene therapy based on extensive preclinical data. However, despite encouraging positive results in early phase clinical trials, more recent larger trials reported only neutral results. Nevertheless, the field has gained important knowledge from these trials and is leading to the development of more cardiotropic vectors and improved delivery systems. It has become more evident that humans are more resistant to therapeutic transgene expression compared to experimental animals and thus refinement in gene delivery tools and methods are essential for future success. We provide an overview of the current status of cardiac gene therapy focusing on gene delivery tools and methods. Newer technologies, devices, and approaches will undoubtedly lead to more promising clinical results in the near future.
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY, 10029-6574, USA.
authors.forename: Kiyotake
authors.initials: K
authors.lastname: Ishikawa
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY, 10029-6574, USA. roger.hajjar@mssm.edu.
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Hajjar
mesh_terms: D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000378:metabolism; D006321:Heart / Q000502:physiology*; D006801:Humans; D011401:Promoter Regions, Genetic / Q000235:genetics
keywords: Adeno-associated vectors; Cardiac gene therapy; Cardiotropic; Gene delivery; Heart failure; Percutaneous delivery; Promoters; Surgical delivery