Moving forward: Cystic Fibrosis Gene Therapy.

Source

Department of Gene Therapy, National Heart and Lung Institute, Imperial College London, and The UK Cystic Fibrosis Gene Therapy Consortium.

Abstract

Since cloning of the CFTR gene more than 20 years ago a large number of pre-clinical and clinical CF gene therapy studies have been performed and a vast amount of information and know-how has been generated. Here, we will review key studies with a particular emphasis on clinical findings. We have learnt that the lung is a more difficult target than originally anticipated and we will describe the strength and weaknesses of the most commonly used airway gene transfer agents. In our view one of the most significant developments in recent years is the generation of lentiviral vectors which efficiently transduce lung tissue. However, focused and co-ordinated efforts assessing lentiviral vector safety and scaling up of production will be required to move this vector into clinical lung gene therapy studies.

TimAllenMDJD

Wednesday, August 7, 2013