Repository logo

Infoscience

  • English
  • French
Log In
Logo EPFL, École polytechnique fédérale de Lausanne

Infoscience

  • English
  • French
Log In
  1. Home
  2. Academic and Research Output
  3. Journal articles
  4. Biosafety in ex vivo gene therapy and conditional ablation of lentivirally transduced hepatocytes in nonhuman primates
 
research article

Biosafety in ex vivo gene therapy and conditional ablation of lentivirally transduced hepatocytes in nonhuman primates

Menzel, Olivier
•
Birraux, Jacques
•
Wildhaber, Barbara E.
Show more
2009
Molecular therapy : the journal of the American Society of Gene Therapy

Ex vivo gene therapy is an interesting alternative to orthotopic liver transplantation (OLT) for treating metabolic liver diseases. In this study, we investigated its efficacy and biosafety in nonhuman primates. Hepatocytes isolated from liver lobectomy were transduced in suspension with a bicistronic liver-specific lentiviral vector and immediately autotransplanted (SLIT) into three cynomolgus monkeys. The vector encoded cynomolgus erythropoietin (EPO) and the conditional suicide gene herpes simplex virus-thymidine kinase (HSV-TK). Survival of transduced hepatocytes and vector dissemination were evaluated by detecting transgene expression and vector DNA. SLIT was safely performed within a day in all three subjects. Serum EPO and hematocrit rapidly increased post-SLIT and their values returned to baseline within about 1 month. Isoforms of EPO detected in monkeys' sera differed from the physiological renal EPO. In liver biopsies at months 8 and 15, we detected EPO protein, vector mRNA and DNA, demonstrating long-term survival and functionality of transplanted lentivirally transduced hepatocytes. Valganciclovir administration resulted in complete ablation of the transduced hepatocytes. We demonstrated the feasibility and biosafety of SLIT, and the long term (>1 year) functionality of lentivirally transduced hepatocytes in nonhuman primates. The HSV-TK/valganciclovir suicide strategy can increase the biosafety of liver gene therapy protocols by safely and completely ablating transduced hepatocytes on demand.

  • Details
  • Metrics
Type
research article
DOI
10.1038/mt.2009.143
Web of Science ID

WOS:000270851900013

Author(s)
Menzel, Olivier
Birraux, Jacques
Wildhaber, Barbara E.
Jond, Caty
Lasne, Françoise
Habre, Walid
Trono, Didier  
Nguyen, Tuan H.
Chardot, Christophe
Date Issued

2009

Publisher

Elsevier

Published in
Molecular therapy : the journal of the American Society of Gene Therapy
Volume

17

Issue

10

Start page

1754

End page

60

Subjects

In-Vivo

•

Stable Transduction

•

Vector Particles

•

Liver-Disease

•

Suicide Gene

•

Transplantation

•

Cells

•

Model

•

Immunodeficiency

•

Integration

Editorial or Peer reviewed

NON-REVIEWED

Written at

OTHER

EPFL units
LVG  
Available on Infoscience
April 26, 2010
Use this identifier to reference this record
https://infoscience.epfl.ch/handle/20.500.14299/49721
Logo EPFL, École polytechnique fédérale de Lausanne
  • Contact
  • infoscience@epfl.ch

  • Follow us on Facebook
  • Follow us on Instagram
  • Follow us on LinkedIn
  • Follow us on X
  • Follow us on Youtube
AccessibilityLegal noticePrivacy policyCookie settingsEnd User AgreementGet helpFeedback

Infoscience is a service managed and provided by the Library and IT Services of EPFL. © EPFL, tous droits réservés