Academic Journal

Gene therapy during ex situ heart perfusion:a new frontier in cardiac regenerative medicine?

Bibliographic Details
Title: Gene therapy during ex situ heart perfusion:a new frontier in cardiac regenerative medicine?
Authors: Vervoorn, Mats T., Amelink,Jantijn J.G.J., Ballan, Elisa M., Doevendans, Pieter A., Sluijter, Joost P.G., Mishra, Mudit, Boink,Gerard J.J., Bowles,Dawn E., van der Kaaij, Niels P., Informatisering, Arts Assistenten CTC, Secretariaat en overig CTC, Experimental Cardiology Laboratory, Innovatie CTC, Cardiologie, Team Medisch, Circulatory Health, Regenerative Medicine and Stem Cells, Onderzoek Cardiovasculair Reg. Med., Experimentele Afd. Cardiologie 2, CTC
Publication Year: 2023
Subject Terms: ex situ heart perfusion, gene therapy, heart failure, heart transplantation (HTx), regenerative medicine, Cardiology and Cardiovascular Medicine, Journal Article, Review
Description: Ex situ organ preservation by machine perfusion can improve preservation of organs for transplantation. Furthermore, machine perfusion opens up the possibilities for selective immunomodulation, creation of tolerance to ischemia-reperfusion injury and/or correction of a pathogenic genetic defect. The application of gene modifying therapies to treat heart diseases caused by pathogenic mutations during ex situ heart perfusion seems promising, especially given the limitations related to delivery of vectors that were encountered during clinical trials using in vivo cardiac gene therapy. By isolating the heart in a metabolically and immunologically favorable environment and preventing off-target effects and dilution, it is possible to directly control factors that enhance the success rate of cardiac gene therapy. A literature search of PubMed and Embase databases was performed to identify all relevant studies regarding gene therapy during ex situ heart perfusion, aiming to highlight important lessons learned and discuss future clinical prospects of this promising approach.
Document Type: article in journal/newspaper
File Description: text/plain
Language: English
ISSN: 2297-055X
Relation: https://dspace.library.uu.nl/handle/1874/451001
Availability: https://dspace.library.uu.nl/handle/1874/451001
Rights: info:eu-repo/semantics/OpenAccess
Accession Number: edsbas.3E12B4E1
Database: BASE
Description
ISSN:2297055X