Liver-directed lentiviral gene therapy in a dog model of hemophilia B-Reference-Cited by-同舟云学术

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Published:2015-03-04 Issue:277 Volume:7 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Cantore Alessio¹²,Ranzani Marco¹²,Bartholomae Cynthia C.³,Volpin Monica¹²,Valle Patrizia Della⁴,Sanvito Francesca⁵,Sergi Lucia Sergi¹,Gallina Pierangela¹,Benedicenti Fabrizio¹,Bellinger Dwight⁶,Raymer Robin⁶,Merricks Elizabeth⁶,Bellintani Francesca⁷,Martin Samia⁸,Doglioni Claudio⁵,D’Angelo Armando⁴,VandenDriessche Thierry⁹¹⁰,Chuah Marinee K.⁹¹⁰,Schmidt Manfred³,Nichols Timothy⁶,Montini Eugenio¹,Naldini Luigi¹²

Affiliation:

1. San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy.

2. Vita-Salute San Raffaele University, 20132 Milan, Italy.

3. Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany.

4. Coagulation Service and Thrombosis Research Unit, San Raffaele Scientific Institute, 20132 Milan, Italy.

5. Pathology Unit, Department of Oncology, San Raffaele Scientific Institute, 20132 Milan, Italy.

6. Department of Pathology, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.

7. MolMed S.p.A., 20132 Milan, Italy.

8. Généthon, 91000 Évry, France.

9. Department of Gene Therapy and Regenerative Medicine, Free University of Brussels, 1050 Brussels, Belgium.

10. Department of Cardiovascular Sciences, Center for Molecular and Vascular Biology, University of Leuven, 3000 Leuven, Belgium.

Abstract

Gene therapy with lentiviral vectors targeting transgene expression to hepatocytes provides stable reconstitution of clotting activity in dogs with hemophilia B and does not show genotoxicity in tumor-prone mice.

Funder

NIH

European Union Seventh Framework Programme

European Research Council Advanced

Telethon

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference51 articles.

1. Haemophilias A and B

2. Current status of haemophilia gene therapy