From Bench to Bedside: Preclinical Evaluation of a Self-Inactivating Gammaretroviral Vector for the Gene Therapy of X-linked Chronic Granulomatous Disease-Reference-Cited by-同舟云学术

From Bench to Bedside: Preclinical Evaluation of a Self-Inactivating Gammaretroviral Vector for the Gene Therapy of X-linked Chronic Granulomatous Disease

Published:2013-06 Issue:2 Volume:24 Page:86-98
ISSN:2324-8637
Container-title:Human Gene Therapy Clinical Development
language:en
Short-container-title:Human Gene Therapy Clinical Development

Author:

Stein Stefan¹,Scholz Simone²,Schwäble Joachim¹³,Sadat Mohammed A.⁴,Modlich Ute⁵,Schultze-Strasser Stephan¹,Diaz Margarita¹,Chen-Wichmann Linping¹,Müller-Kuller Uta¹,Brendel Christian¹,Fronza Raffaele²,Kaufmann Kerstin B.¹,Naundorf Sonja⁶,Pech Nancy K.⁴,Travers Jeffrey B.⁷,Matute Juan D.⁴,Presson Robert G.⁸,Sandusky George E.⁹,Kunkel Hana¹,Rudolf Eva¹,Dillmann Adelina¹,von Kalle Christof²,Kühlcke Klaus⁶,Baum Christopher⁵,Schambach Axel⁵,Dinauer Mary C.,Schmidt Manfred²,Grez Manuel¹

Affiliation:

1. Institute for Biomedical Research, Georg-Speyer-Haus, 60596 Frankfurt, Germany.

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

3. Department of Hematology/Oncology, Johann Wolfgang Goethe University, 60590 Frankfurt, Germany.

4. Herman B. Wells Center for Pediatric Research, Department of Pediatrics, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana.

5. Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.

6. European Institute for Research and Development of Transplantation Strategies AG, 55743 Idar-Oberstein, Germany.

7. Department of Dermatology, Indiana University School of Medicine, 46202 Indianapolis, Indiana.

8. Department of Anesthesia, Indiana University School of Medicine, 46202 Indianapolis, Indiana.

9. Department of Pathology and Laboratory Medicine, Indiana University School of Medicine, 46202 Indianapolis, Indiana.

Publisher

Mary Ann Liebert Inc

Subject

Genetics(clinical)

Link

http://www.liebertpub.com/doi/pdf/10.1089/humc.2013.019

Reference61 articles.

1. Gene therapy for primary immunodeficiencies: Part 2

2. Development of Novel Efficient SIN Vectors with Improved Safety Features for Wiskott–Aldrich Syndrome Stem Cell Based Gene Therapy

3. Chance or necessity? Insertional Mutagenesis in Gene Therapy and Its Consequences

4. Concise Review: Managing Genotoxicity in the Therapeutic Modification of Stem Cells

5. Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

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