Gene Therapy: The First Approved Gene-Based Medicines, Molecular Mechanisms and Clinical Indications

Title: Gene Therapy: The First Approved Gene-Based Medicines, Molecular Mechanisms and Clinical Indications

Volume: 1 Issue: 1

Author(s): J. K. Raty, J. T. Pikkarainen, T. Wirth and S. Yla-Herttuala

Affiliation:

Keywords: Adenovirus, p53, conditional replication, thymidine kinase

Abstract: 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.

Cite this article as:

Raty K. J., Pikkarainen T. J., Wirth T. and Yla-Herttuala S., Gene Therapy: The First Approved Gene-Based Medicines, Molecular Mechanisms and Clinical Indications, Current Molecular Pharmacology 2008; 1(1) . https://dx.doi.org/10.2174/1874467210801010013