title: Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human.
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.
authors.affiliation: Faculty of Medicine, Imperial College London, London, UK.
authors.forename: S E
authors.initials: SE
authors.lastname: Khorsandi
authors.forename: P
authors.initials: P
authors.lastname: Bachellier
authors.forename: J C
authors.initials: JC
authors.lastname: Weber
authors.forename: M
authors.initials: M
authors.lastname: Greget
authors.forename: D
authors.initials: D
authors.lastname: Jaeck
authors.forename: D
authors.initials: D
authors.lastname: Zacharoulis
authors.forename: C
authors.initials: C
authors.lastname: Rountas
authors.forename: S
authors.initials: S
authors.lastname: Helmy
authors.forename: A
authors.initials: A
authors.lastname: Helmy
authors.forename: M
authors.initials: M
authors.lastname: Al-Waracky
authors.forename: H
authors.initials: H
authors.lastname: Salama
authors.forename: L
authors.initials: L
authors.lastname: Jiao
authors.forename: J
authors.initials: J
authors.lastname: Nicholls
authors.forename: A J
authors.initials: AJ
authors.lastname: Davies
authors.forename: N
authors.initials: N
authors.lastname: Levicar
authors.forename: S
authors.initials: S
authors.lastname: Jensen
authors.forename: N
authors.initials: N
authors.lastname: Habib
mesh_terms: D000328:Adult; D000818:Animals; D005260:Female; D015316:Genetic Therapy; D006801:Humans; D008103:Liver Cirrhosis / Q000150:complications / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D010957:Plasmids; D013552:Swine; D013921:Thrombocytopenia / Q000150:complications / Q000628:therapy*; D013926:Thrombopoietin / Q000235:genetics
chemical_list: D013926:Thrombopoietin