Gene Therapy Protocols: Production and In Vivo Applications of Gene Transfer Vectors

386 Pages·2008·4.339 MB·English

by  Joseph M. Le Doux (auth.)

Gene Therapy Protocols
M E T H O D S I N M O L E C U L A R B I O L O G YTM
John M. Walker, SERIES EDITOR
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M E T H O D S I N M O L E C U L A R B I O L O G YTM
Gene Therapy
Protocols
Volume 1: Production and In Vivo Applications
of Gene Transfer Vectors
Third Edition
Edited by
Joseph M. Le Doux
Department of Biomedical Engineering, Georgia Institute
of Technology and Emory University, Atlanta, GA
Editor
JosephM.LeDoux
DepartmentofBiomedicalEngineering
GeorgiaInstituteofTechnologyandEmoryUniversity
Atlanta,Georgia30322-0355
[email protected]
SeriesEditor
JohnM.Walker
SchoolofLifeSciences
UniversityofHertfordshire
CollegeLane
HatfieldCampus
Hatfield,Herts.,UKAL109AB
[email protected]
ISBN:978-1-58829-903-1 e-ISBN:978-1-59745-237-3
ISSN:1064-3745 e-ISSN:1940-6029
LibraryofCongressControlNumber:2007941274
©2008HumanaPress,apartofSpringerScience+BusinessMedia,LLC
All rights reserved. This work may not be translated or copied in whole or in part without the written
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Cover illustration: Background image: Photo provided courtesy of Dongsheng Duan. Modified by Nancy
Fallatt with permission. Cover image: Figure 1, Chapter 10. “Non-Viral Gene Delivery with Cationic
Liposome-DNA Complexes,” by Kai K. Ewert, Ayesha Ahmad, Nathan F. Bouxsein, Heather M. Evans,
and Cyrus R. Safinya. Reprinted (abstracted/excerpted) with permission from J. Am. Chem. Soc. 2006,
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Preface
Gene therapy has the potential to significantly impact human healthcare in the
twenty-first century. The idea behind gene therapy is simple: to deliver genetic
material to cells that will slow down or halt the progression of disease, or to
help repair or regenerate damaged or lost tissues. To successfully implement
this simple idea, however, we must first address a number of technological
challenges. One such challenge is the production of gene transfer vectors that
can safely and effectively transfer genes to a wide variety of cells, tissues,
and organs. A number of vectors have been developed, but none are ideal.
The choice of which system to use depends on the specific gene therapy
application being addressed, and is often influenced by its specific functional
characteristics, such as the types of cells it can transduce, the efficiency with
which it transfers genes, the strength and persistence of transgene expression,
and the extent to which it activates the immune response of the host. For all
genetransfervectorsystems,itiscriticalthatproductionmethodsaredeveloped
that are economical, that can be conducted on a large scale, and that yield
a high-titer product that does not require extensive postproduction processing
or purification. Another critical issue is the development of clinically relevant
protocols for using these gene transfer vector systems. Numerous experimental
protocols have been developed over the past few years. These protocols share
a number of common elements, but they also each harbor their own share
of unique technical challenges and issues that were addressed and overcome
through years of hard work, creativity, and perseverance. These hard-earned
protocols can sometimes be used “as is” by others if they are addressing the
same or similar gene therapy application, or they can serve as the basis for the
development of new protocols.
The field of gene therapy is rapidly advancing on all of these fronts. Signif-
icant improvements have been made in the methods used to produce the core
gene transfer technologies. In parallel, novel gene transfer protocols have been
developed that have helped to expand the list of potential applications of gene
therapy.Inthisnewandentirelyrevisedthirdedition,GeneTherapyProtocols,
Volumes 1 and 2 present a comprehensive collection of detailed methods and
protocols used by the leaders in the field of gene therapy. The first volume
covers current and emerging methods for the production of major viral and
nonviral gene transfer vectors. The second volume provides detailed protocols
for some of the most important applications of gene therapy, including the
v
vi Preface
areas of stem cell biology, cancer, diabetes, HIV, and tissue engineering. This
compilationofprotocolsisexpectedtoserveasavaluableresourceforgraduate
students and postdoctoral fellows, as well as for basic and clinical researchers
in the industry and academia.
Joseph M. LeDoux
Contents
Preface......................................................... v
Contributors.................................................... ix
1. Preparation and Quantification of Pseudotyped Retroviral
Vector....................................................... 1
Hong Yu and Young Jik Kwon
2. Production of Retroviral Vectors for Clinical Use................. 17
Kenneth Cornetta, Lilith Reeves, and Scott Cross
3. Methods for the Production of Helper-Dependent Adenoviral
Vectors....................................................... 33
Donna J. Palmer and Philip Ng
4. Methods for the Production of First Generation Adenoviral
Vectors....................................................... 55
Donna J. Palmer and Philip Ng
5. Large-Scale Production of Recombinant Adeno-Associated Viral
Vectors....................................................... 79
Alejandro Negrete and Robert M. Kotin
6. Construction and Production of Recombinant Herpes Simplex
Virus Vectors................................................. 97
William F. Goins, David M. Krisky, James B. Wechuck,
Shaohua Huang, and Joseph C. Glorioso
7. Plasmid-Based Gene Transfer in Mouse Skeletal Muscle by
Electroporation............................................... 115
Jonathan D. Schertzer and Gordon S. Lynch
8. Chitosan Nanoparticle-Mediated Gene Transfer.................. 127
Dongwon Lee and Shyam S. Mohapatra
9. PEG–PEI Copolymers for Oligonucleotide Delivery to Cells
and Tissues................................................... 141
Gordon J. Lutz, Shashank R. Sirsi, and Jason H. Williams
10. Non-Viral Gene Delivery with Cationic Liposome–DNA
Complexes................................................... 159
Kai K. Ewert, Ayesha Ahmad, Nathan F. Bouxsein,
Heather M. Evans, and Cyrus R. Safinya
vii
viii Contents
11. Applications of Lentiviral Vectors in Noninvasive Molecular
Imaging...................................................... 177
Abhijit De, Shahriar Shah Yaghoubi, and Sanjiv Sam Gambhir
12. Retroviral Modification of Mesenchymal Stem Cells for Gene
Therapy of Hemophilia A..................................... 203
Christopher B. Doering
13. Transduction of Murine Hematopoietic Stem Cells and In Vivo
Selection of Gene-Modified Cells............................. 213
Lucienne M. Ide, Elisabeth Javazon, and H. Trent Spencer
14. Assessment of CFTR Function after Gene Transfer In Vitro
and In Vivo.................................................. 229
Uta Griesenbach, Felix M. Munkonge, Stephanie Sumner-Jones,
Emma Holder, Stephen N. Smith, A. Christopher Boyd,
Deborah R. Gill, Stephen C. Hyde, David Porteous,
and Eric W. F. W. Alton; on behalf of the UK Cystic Fibrosis
Gene Therapy Consortium
15. Oncolytic Adenoviruses for Cancer Gene Therapy............... 243
Ta-Chiang Liu, Stephen H. Thorne, and David H. Kirn
16. Design of Trans-Splicing Adeno-Associated Viral Vectors
for Duchenne Muscular Dystrophy Gene Therapy............. 259
Yi Lai, Dejia Li, Yongping Yue, and Dongsheng Duan
17. Prevention of Type 1 Diabetes in NOD Mice by Genetic
Engineering of Hematopoietic Stem Cells...................... 277
Jessamyn Bagley, Chaorui Tian, and John Iacomini
18. Lentiviral Vector Delivery of siRNA and shRNA Encoding Genes
into Cultured and Primary Hematopoietic Cells................ 287
Mingjie Li and John J. Rossi
19. Nanoparticle-Mediated Gene Delivery to the Lung............... 301
Rajagopal Ramesh
20. Retroviral-Mediated Gene Therapy for the Differentiation
of Primary Cells into a Mineralizing Osteoblastic Phenotype... 333
Jennifer E. Phillips and Andrés J. García
21. In Vivo siRNA Delivery to the Mouse Hypothalamus Shows a Role
of the Co-Chaperone XAP2 in Regulating TRH
Transcription................................................. 355
Stéphanie Decherf, Zahra Hassani, and Barbara A. Demeneix
22. Efficient Retroviral Gene Transfer to Epidermal Stem Cells........ 367
Pedro Lei and Stelios T. Andreadis
Index........................................................... 381
Contributors
Ayesha Ahmad • Materials Department, Physics Department and
Molecular, Cellular, and Developmental Biology Department, University
of California, Santa Barbara, CA
Eric W. F. W. Alton • Department of Gene Therapy, Faculty of Medicine,
Imperial College London, UK, and Medical Genetics, Molecular Medicine
Centre, University of Edinburgh, Edinburgh, UK
Stelios T. Andreadis • Department of Chemical and Biological
Engineering, University of Buffalo, The State University of New York,
Buffalo, NY
Jessamyn Bagley • Transplantation Research Center, Renal Division,
Brigham and Women’s Hospital and Children’s Hospital Boston, Harvard
Medical School, Boston, MA
Nathan F. Bouxsein • Materials Department, Physics Department and
Molecular, Cellular, and Developmental Biology Department, University
of California, Santa Barbara, CA
A. Christopher Boyd • The UK Cystic Fibrosis Gene Therapy Consortium
and Medical Genetics, Molecular Medicine Centre, University of
Edinburgh, Edinburgh, UK
Kenneth Cornetta • Department of Medical and Molecular Genetics,
Indiana University School of Medicine, Indianapolis, IN
Scott Cross • Department of Medical and Molecular Genetics, Indiana
University School of Medicine, Indianapolis, IN
Abhijit De • Molecular Imaging Program at Stanford (MIPS) and Bio-X
Program, Departments of Radiology and Bioengineering, School of
Medicine, Stanford University, Stanford, CA
Stéphanie Decherf • Laboratoire d’Evolution des Régulations
Endocriniennes, Muséum National d’Histoire, Naturelle, Paris, France
Barbara A. Demeneix • Laboratoire d’Evolution des Régulations
Endocriniennes, Muséum National d’Histoire, Naturelle, Paris, France
Christopher B. Doering • Aflac Cancer Center and Blood Disorders
Service, Department of Pediatrics, Emory University, Atlanta, GA
Dongsheng Duan • Department of Molecular Microbiology and
Immunology, University of Missouri, Columbia, MO
ix
x Contributors
Heather M. Evans • Materials Department, Physics Department and
Molecular, Cellular, and Developmental Biology Department, University
of California, Santa Barbara, CA
Kai K. Ewert • Materials Department, Physics Department and Molecular,
Cellular, and Developmental Biology Department, University of California,
Santa Barbara, CA
Sanjiv Sam Gambhir • Molecular Imaging Program at Stanford (MIPS) and
Bio-X Program, Departments of Radiology and Bioengineering, School of
Medicine, Stanford University, Stanford, CA
Andrés J. García • Petit Institute for Bioengineering and Biosciences and
Georgia Tech/Emory Center for the Engineering of Living Tissues, Georgia
Institute of Technology, Atlanta, GA
Deborah R. Gill • The UK Cystic Fibrosis Gene Therapy Consortium and
Gene Medicine Group, University of Oxford, Oxford, UK
Joseph C. Glorioso • Department of Molecular Genetics and Biochemistry,
University of Pittsburgh School of Medicine, Pittsburgh, PA
William F. Goins • Department of Molecular Genetics and Biochemistry,
University of Pittsburgh School of Medicine, Pittsburgh, PA
Uta Griesenbach • Department of Gene Therapy, Faculty of Medicine,
Imperial College London, UK, and The UK Cystic Fibrosis Gene Therapy
Consortium, London, UK
Zahra Hassani • Laboratoire d’Evolution des Régulations Endocriniennes,
Muséum National d’Histoire Naturelle, CNRS, Paris, France
Emma Holder • Medical Genetics, Molecular Medicine Centre, University
of Edinburgh, Edinburgh, UK, and The UK Cystic Fibrosis Gene Therapy
Consortium, London, UK
Shaohua Huang • Department of Molecular Genetics and Biochemistry,
University of Pittsburgh School of Medicine, Pittsburgh, PA
Stephen C. Hyde • The UK Cystic Fibrosis Gene Therapy Consortium and
Gene Medicine Group, University of Oxford, Oxford, UK
John Iacomini • Transplantation Research Center, Renal Division Brigham
and Women’s Hospital and Children’s Hospital Boston, Harvard Medical
School, Boston, MA
Lucienne M. Ide • Aflac Cancer Center and Blood Disorders Service,
Division of Hematology/Oncology and Bone Marrow Transplantation,
Department of Pediatrics, Emory University School of Medicine, Atlanta,
GA
Elisabeth Javazon • Aflac Cancer Center and Blood Disorders Service,
Division of Hematology/Oncology and Bone Marrow Transplantation,
Department of Pediatrics, Emory University School of Medicine, Atlanta,
GA