Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1-Reference-Cited by-同舟云学术

Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1

Published:2016-07-27 Issue:349 Volume:8 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Hickey Raymond D.¹²,Mao Shennen A.¹,Glorioso Jaime¹,Elgilani Faysal¹,Amiot Bruce³,Chen Harvey¹,Rinaldo Piero⁴,Marler Ronald⁵,Jiang Huailei⁶,DeGrado Timothy R.⁶,Suksanpaisan Lukkana⁶⁷,O’Connor Michael K.⁶,Freeman Brittany L.⁸,Ibrahim Samar H.⁸,Peng Kah Whye²,Harding Cary O.⁹,Ho Chak-Sum¹⁰,Grompe Markus¹¹,Ikeda Yasuhiro²,Lillegard Joseph B.¹¹²,Russell Stephen J.²,Nyberg Scott L.¹

Affiliation:

1. Department of Surgery, Mayo Clinic, Rochester, MN 55905, USA.

2. Department of Molecular Medicine, Mayo Clinic, Rochester, MN 55905, USA.

3. Brami Biomedical Inc., Coon Rapids, MN 55433, USA.

4. Division of Laboratory Genetics, Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN 55905, USA.

5. Department of Comparative Medicine, Mayo Clinic, Scottsdale, AZ 85259, USA.

6. Department of Radiology, Mayo Clinic, Rochester, MN 55905, USA.

7. Imanis Life Sciences, Rochester, MN 55902, USA.

8. Division of Pediatric Gastroenterology, Mayo Clinic, Rochester, MN 55905, USA.

9. Department of Molecular and Medical Genetics and Department of Pediatrics, Oregon Health and Science University, Portland, OR 97239, USA.

10. Histocompatibility Laboratory, Gift of Life Michigan, Ann Arbor, MI 48108, USA.

11. Papé Family Pediatric Research Institute, Oregon Health and Science University, Portland, OR 97239, USA.

12. Midwest Fetal Care Center, Children’s Hospitals and Clinics of Minnesota, Minneapolis, MN 55404, USA.

Abstract

Transplantation of gene-corrected autologous hepatocytes can cure metabolic disease in a preclinical pig model of hereditary tyrosinemia type 1.

Funder

American Liver Foundation

Mayo Clinic Center for Regenerative Medicine Career Development Award

NIH

Wallace H. Coulter Foundation

Marriot Foundation

Darwin Deason Family Foundation

Mayo Foundation

Center for Clinical and Translational Science KL2

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference45 articles.

1. On the enzymic defects in hereditary tyrosinemia.

2. HOMOGENTISATE METABOLISM: THE ISOMERIZATION OF MALEYLACETOACETATE BY AN ENZYME WHICH REQUIRES GLUTATHIONE

3. Tyrosinaemia type I and apoptosis of hepatocytes and renal tubular cells

4. Hepatocyte injury in tyrosinemia type 1 is induced by fumarylacetoacetate and is inhibited by caspase inhibitors

5. The Mutagenicity of the Tyrosine Metabolite, Fumarylacetoacetate, Is Enhanced by Glutathione Depletion

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