Gene therapy: the first approved gene-based medicines, molecular mechanisms and clinical indications.
As gene therapy has matured from clinical trials to the first commercial products, understanding of the mechanisms of gene delivery has increased tremendously. This has also been reflected in viral vector development, creating a number of new approaches to tackle issues in transduction efficiency, biodistribution and viral safety. This review will highlight the most important issues and advancements in vector development, administration, surface modification, integration to host genome and safety. The gene therapy products currently available or near market approval, based on p53 expression (Gendicine and Advexin), conditionally replicative adenoviruses (Oncorine) and thymidine kinase + ganciclovir therapy (Cerepro), are introduced with emphasis on the molecular mechanisms of action.
Räty
J K
JK
Department of Biotechnology and Molecular Medicine, A.I. Virtanen Institute for Molecular Sciences, University of Kuopio, Finland. jani.raty@uku.fi
Pikkarainen
J T
JT
Wirth
T
T
Ylä-Herttuala
S
S
D000256:Adenoviridae / Q000235:genetics; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000276:immunology; D006801:Humans; D050504:Oncolytic Viruses / Q000235:genetics; D013937:Thymidine Kinase / Q000235:genetics / Q000494:pharmacology; D016159:Tumor Suppressor Protein p53 / Q000235:genetics / Q000494:pharmacology
D016159:Tumor Suppressor Protein p53; D013937:Thymidine Kinase