Optimizing gene delivery vectors for the treatment of heart disease.
BACKGROUND
Cardiac gene therapy is approaching reality, with clinical trials entering Phase II/III. Even so, challenges exist to improve the efficacy of even the most successful therapies.


OBJECTIVE
The merits of different gene therapy vectors are weighed to assess the current feasibility of each in specific cardiac applications. Major obstacles are discussed, along with recent advances in vector development to overcome or circumvent those difficulties.


METHODS
This review focuses primarily on gene delivery via naked DNA, adenovirus, lentivirus, and adeno-associated virus (AAV) vectors.


CONCLUSIONS
Gene therapy via adenovirus and AAV vectors has developed into a promising option for the treatment of heart disease. The merits of gene therapy compared with emerging stem cell and microRNA-based treatments are discussed.
Gray
Steven J
SJ
University of North Carolina at Chapel Hill, Gene Therapy Center, Chapel Hill, North Carolina 27599, USA.
Samulski
R Jude
RJ
D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D002986:Clinical Trials as Topic; D000229:Dependovirus / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006331:Heart Diseases / Q000628:therapy*; D006801:Humans; D007700:Kinetics; D016086:Lentivirus / Q000235:genetics; D035683:MicroRNAs / Q000378:metabolism; D011401:Promoter Regions, Genetic; D013234:Stem Cells / Q000166:cytology; D019076:Transgenes
D035683:MicroRNAs