Surgical Methods for Cardiac Gene Delivery in Large Animals.
Cardiac gene therapy
Cardiopulmonary bypass
Cardiovascular diseases
Gene delivery
Intramyocardial administration
Journal
Methods in molecular biology (Clifton, N.J.)
ISSN: 1940-6029
Titre abrégé: Methods Mol Biol
Pays: United States
ID NLM: 9214969
Informations de publication
Date de publication:
2022
2022
Historique:
entrez:
30
8
2022
pubmed:
31
8
2022
medline:
3
9
2022
Statut:
ppublish
Résumé
This chapter describes main strategies of surgical gene delivery in large animals. Existing methods of cardiac gene transfer can be classified by the site of injection, interventional approach, and type of cardiac circulation at the time of transfer. Randomized clinical trials have suggested that the therapeutic benefits of gene therapy are not as substantial as expected from animal studies. This discordance in results is largely due to gene delivery methods that may be effective in small animals but are not scalable to larger species and, therefore, cannot transduce a sufficient fraction of myocytes to establish long-term clinical efficacy. Ideally, an optimized gene transfer should incorporate the following: a closed-loop recirculation for extended transgene residence time; vector washout form the vascular system after transfer to prevent collateral expression; use of methods to increase myocardial transcapillary gradient for viral particles for a better transduction, probably retrograde route of gene delivery through the coronary venous system; and myocardial ischemic preconditioning.
Identifiants
pubmed: 36040596
doi: 10.1007/978-1-0716-2707-5_15
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
189-203Informations de copyright
© 2022. The Author(s), under exclusive license to Springer Science+Business Media, LLC, part of Springer Nature.
Références
Lin H, Parmacek MS, Morle G et al (1990) Expression of recombinant genes in myocardium in vivo after injection of DNA. Circulation 82:2217–2221
doi: 10.1161/01.CIR.82.6.2217
Guzman RJ, Lemarchand P, Crystal RG et al (1993) Efficient gene transfer into myocardium by direct injection of adenovirus vectors. Circ Res 73:1202–1207
doi: 10.1161/01.RES.73.6.1202
Barr E, Carroll J, Kalynych AM et al (1994) Efficient catheter-mediated gene transfer into the heart using replication-defective adenovirus. Gene Ther 1:51–58
Ishikawa K, Tilemann L, Ladage D et al (2012) Cardiac gene therapy in large animals: bridge from bench to bedside. Gene Ther 19:670–677
doi: 10.1038/gt.2012.3
Katz MG, Fargnoli AS, Williams RD et al (2015) Cell and gene therapies for cardiovascular disease. In: Templeton NS (ed) Gene and cell therapy. CRC Press, New York, pp 861–901
doi: 10.1201/b18002-39
Ishikawa K, Weber T, Hajjar RJ (2018) Human cardiac gene therapy. Circ Res 123:601–613
doi: 10.1161/CIRCRESAHA.118.311587
Katz MG, Fargnoli AS, Weber T et al (2017) Use of adeno-associated virus vector for cardiac gene delivery in large-animal surgical models of heart failure. Hum Gene Ther Clin Dev 28:157–164
doi: 10.1089/humc.2017.070
Ishikawa K, Tilemann L, Fish K et al (2011) Gene delivery methods in cardiac gene therapy. J Gene Med 13:566–572
doi: 10.1002/jgm.1609
Katz MG, Fargnoli AS, Williams RD et al (2013) Gene therapy delivery systems for enhancing viral and nonviral vectors for cardiac diseases: current concepts and future applications. Hum Gene Ther 24:914–927
doi: 10.1089/hum.2013.2517
Hajjar RJ, Ishikawa K (2017) Introducing genes to the heart: all about delivery. Circ Res 120:33–35
doi: 10.1161/CIRCRESAHA.116.310039
Hulot JS, Ishikawa K, Hajjar RJ (2016) Gene therapy for the treatment of heart failure: promise postponed. Eur Heart J 37:1651–1658
doi: 10.1093/eurheartj/ehw019
Katz MG, Fargnoli AS, Kendle AP et al (2016) Gene therapy in cardiac surgery: clinical trials, challenges, and perspectives. Ann Thorac Surg 101:2407–2416
doi: 10.1016/j.athoracsur.2015.12.004
Katz MG, Swain JD, White JD et al (2010) Cardiac gene therapy: optimization of gene delivery techniques in vivo. Hum Gene Ther 21:371–380
doi: 10.1089/hum.2009.164