Original Article

Cancer Gene Therapy (2008) 15, 225–230; doi:10.1038/sj.cgt.7701119; published online 8 February 2008

Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

S E Khorsandi1, P Bachellier1,2, J C Weber1,2, M Greget2, D Jaeck2, D Zacharoulis3, C Rountas3, S Helmy1,4, A Helmy4, M Al-Waracky4, H Salama4, L Jiao1, J Nicholls1, A J Davies1, N Levicar1, S Jensen1 and N Habib1

  1. 1Faculty of Medicine, Imperial College London, London, UK
  2. 2Foundation Transplantation, Strasbourg, Hôspital Hautpierre, Strasbourg, France
  3. 3Larissa University School of Medicine, Larissa, Greece
  4. 4Faculty of Medicine, Cairo University, Cairo, Egypt

Correspondence: Professor N Habib, Imperial College London, Faculty of Medicine, Hammersmith Hospital, Du Cane Road, London W12 0NN, UK. E-mail: nagy.habib@imperial.ac.uk

Received 17 April 2007; Revised 1 November 2007; Accepted 24 November 2007; Published online 8 February 2008.

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Abstract

This paper highlights our experience of the transfer of hydrodynamic gene therapy (HGT) from the large animal, the pig, into clinical practice. The modification of balloon catheters and the development of a minimally invasive technique to allow selective isolation of liver segments for HGT in the large animal and human are described. Finally, our preliminary results from a phase I clinical study of HGT for thrombopoietin (TPO) in cirrhotic patients with thrombocytopenia are discussed. Based on these provisional data, minimally invasive selective HGT of liver segments appears to be technically safe, but further work is required to optimize the efficiency of gene transfer in order to achieve clinical benefit.

Keywords:

hydrodynamic gene therapy, human, pig

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