title: Methods to improve cardiac gene therapy expression.
abstract: Gene therapy strategies are becoming a valuable approach for the treatment of heart failure. Some trials are ongoing and others are being organized. Vascular access in clinical experimentation is still the chosen modality of delivery, but many other approaches are in research and development. A successful gene therapy strategy involves not only the choice of the right vector and gene, but also the correct delivery strategy that allows for transduction of the highest percentage of cardiomyocytes, limited spilling of virus into other organs and the possibility to correlate the amount of injected virus to the rate of the expression within the cardiac tissue. The authors will first concentrate on clarifying what the barriers are that the virus has to overcome in order to reach the nuclei of the target organs and methodologies that have been tested to improve the range of expression.
authors.affiliation: Temple University, School of Medicine, Philadelphia, Pennsylvania, USA.
authors.forename: Maria Cecilia
authors.initials: MC
authors.lastname: Scimia
authors.forename: Kate E
authors.initials: KE
authors.lastname: Sydnes
authors.forename: Daniel A
authors.initials: DA
authors.lastname: Zuppo
authors.forename: Walter J
authors.initials: WJ
authors.lastname: Koch
mesh_terms: D000818:Animals; D002316:Cardiotonic Agents / Q000627:therapeutic use; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000627:therapeutic use*; D006331:Heart Diseases / Q000628:therapy*; D006801:Humans; D009206:Myocardium / Q000378:metabolism*
chemical_list: D002316:Cardiotonic Agents
keywords: adeno-associated viruses; antegrade injection; cardiomyocytes; clinical usage; electroporation; gene gun-mediated delivery; gene therapy; heart failure; ischemia; jet injection; laser-based transfection; magnetofection; myocardial infarction; retrograde injection; sonoporation; vascular delivery