Safety and Liver Transduction Efficacy of rAAV5-cohPBGD in Nonhuman Primates: A Potential Therapy for Acute Intermittent Porphyria-Reference-Cited by-同舟云学术

Safety and Liver Transduction Efficacy of rAAV5-cohPBGD in Nonhuman Primates: A Potential Therapy for Acute Intermittent Porphyria

Published:2013-12 Issue:12 Volume:24 Page:1007-1017
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Pañeda Astrid¹²,Lopez-Franco Esperanza¹,Kaeppel Christine³,Unzu Carmen¹,Gil-Royo Ana Gloria⁴,D'Avola Delia⁵⁶,Beattie Stuart G.⁷,Olagüe Cristina¹,Ferrero Roberto¹,Sampedro Ana¹,Mauleon Itsaso¹,Hermening Stephan⁷,Salmon Florence⁷,Benito Alberto⁵,Gavira Juan Jose⁵,Cornet María Eugenia²,del Mar Municio María²,von Kalle Christof³,Petry Harald⁷,Prieto Jesus¹⁵⁶,Schmidt Manfred³,Fontanellas Antonio¹,González-Aseguinolaza Gloria¹

Affiliation:

1. Division of Hepatology and Gene Therapy, Center for Applied Medical Research, 31008 Pamplona, Spain.

2. DIGNA Biotech, 28020 Madrid, Spain.

3. National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany.

4. Department of Pharmacology and Toxicology, University of Navarra, (UNAV), 31008 Pamplona, Spain.

5. Department of Radiology, Department of Cardiology, University Clinic, UNAV, 31008 Pamplona, Spain.

6. Liver Unit and CIBERehd, 31008 Pamplona, Spain.

7. UniQure, 1105 BA, Amsterdam, The Netherlands.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2013.166

Reference35 articles.

1. Analyzing the Number of Common Integration Sites of Viral Vectors – New Methods and Computer Programs

2. Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

3. Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

4. Liver transplantation for acute intermittent porphyria is complicated by a high rate of hepatic artery thrombosis

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