JOURNAL ARTICLE

Gene therapy for cardiac diseases: methods, challenges, and future directions.

  • Published In: Cardiovascular Research, 2024, v. 120, n. 14. P. 1664 1 of 3

  • Database: Academic Search Ultimate 2 of 3

  • Authored By: Grisorio, Luca; Bongianino, Rossana; Gianeselli, Matteo; Priori, Silvia Giuliana 3 of 3

Abstract

This article focuses on recent advances and challenges in cardiac gene therapy for inherited arrhythmias and genetic cardiomyopathies. It reviews molecular strategies—including gene transfer, non-coding nucleic acids, and genome editing—and delivery vectors such as adeno-associated viruses (AAVs) and nanoparticles, emphasizing their therapeutic applications and limitations. The article highlights the importance of optimizing vector tropism, promoter specificity, and delivery routes to enhance efficacy and safety, while discussing preclinical models and clinical trials that illustrate both progress and translational hurdles. Notably, evidence suggests that partial transduction of cardiomyocytes may suffice to achieve therapeutic benefit, a concept with significant implications for future gene therapy development in cardiac diseases.

Additional Information

  • Source:Cardiovascular Research. 2024/10, Vol. 120, Issue 14, p1664
  • Document Type:Article
  • Subject Area:Sports and Leisure
  • Publication Date:2024
  • ISSN:0008-6363
  • DOI:10.1093/cvr/cvae207
  • Accession Number:181894239
  • Copyright Statement:Copyright of Cardiovascular Research is the property of Oxford University Press / USA and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

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