Steven J Gray1, R Jude Samulski. 1. University of North Carolina at Chapel Hill, Gene Therapy Center, Chapel Hill, North Carolina 27599, USA.
Abstract
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. CONCLUSION: 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.
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. CONCLUSION: 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.
Authors: Mohammad A Mandegar; Daniela Moralli; Suhail Khoja; Sally Cowley; David Y L Chan; Mohammed Yusuf; Sayandip Mukherjee; Michael P Blundell; Emanuela V Volpi; Adrian J Thrasher; William James; Zoia L Monaco Journal: Hum Mol Genet Date: 2011-05-18 Impact factor: 6.150
Authors: Lars Maegdefessel; Junya Azuma; Ryuji Toh; Denis R Merk; Alicia Deng; Jocelyn T Chin; Uwe Raaz; Anke M Schoelmerich; Azad Raiesdana; Nicholas J Leeper; Michael V McConnell; Ronald L Dalman; Joshua M Spin; Philip S Tsao Journal: J Clin Invest Date: 2012-01-24 Impact factor: 14.808