Nonviral Vectors for Gene Therapy: Methods and Protocols (Methods in Molecular Medicine)

413 Pages·2001·2.445 MB·English

by  Mark A. Findeis

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Nonviral Vectors for Gene Therapy
M E T H O D S I N M O L E C U L A R M E D I C I N ETM
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 M E D I C I N ETM
Nonviral Vectors
for Gene Therapy
Methods and Protocols
Edited by
Mark A. Findeis
Praecis Pharmaceuticals Incorporated, Waltham, MA
Humana Press Totowa, New Jersey
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Library of Congress Cataloging in Publication Data
Nonviral vectors for gene therapy : methods and protocols / edited by Mark A. Findeis.
p. ; cm. -- (Methods in molecular medicine ; 65)
Includes bibliographical references and index.
ISBN 0-89603-712-6 (alk. paper)
1. Gene therapy--Laboratory manuals. 2. Genetic vectors--Laboratory manuals. I.
Findeis, Mark A. II. Series.
[DNLM: 1. Genetic Vectors--therapeutic use. 2. Drug Delivery Systems. 3. Gene
Therapy--methods. 4. Gene Transfer Techniques. QH 442.2 N8144 2001]
RB155.8 .N665 2001
616.'.042--dc21
2001016933
Preface
The purpose of this volume of Methods in Molecular Medicine is to set
forth examples of the great variety of techniques and applications that are now
emerging in the field of nonviral gene therapy. The book emphasizes not only
specific approaches to gene delivery but, in particular, the best current meth-
ods to prepare, handle, and characterize gene delivery agents. These topics are
of very broad importance since gene therapy evolves from its mostly acad-
emy-based experimental and clinical research to the ever increasing number
of industry-driven programs directed toward commercial development. Suc-
cessful introduction of nonviral gene therapy agents into the clinic should be
expected to require rigorous manufacturing and analytical methods that readily
meet the regulatory guidelines under which new drug candidates are reviewed
for marketing approval. Exactly what those guidelines will prove to be cer-
tainly depends on the established guidelines for review of both biological and
chemical therapeutics. Additionally, many new techniques are being devised
and applied to gene therapy research; these techniques will be instrumental in
developing and characterizing successful gene delivery agents.
Nonviral Vectors for Gene Therapy: Methods and Protocolshas two main
sections. To start with, there is a series of chapters on specific protocols for
the synthesis, characterization, and application of gene delivery agents. Sev-
eral chapters address the topic of materials to bind with DNA to form the
compact condensed phases that facilitate cellular delivery. Variations on this
theme are addressed by using peptide conjugates, synthetic polymers, and lip-
ids. Increasingly refined methods for the characterization of delivery systems
and their complexes with DNA are described both as part of synthesis proto-
cols and as separate topics. One still relatively new analytical technique is
atomic force microscopy; this should rapidly gain attention for its applica-
bility to the characterization of DNA condensation. Subsequent chapters de-
scribe approaches to gene transfer in vivo, including direct delivery by
intratumoral injection or indirect delivery by cell-specific targeting of DNA
complexes.
The latter section of Nonviral Vectors for Gene Therapy: Methods and
Protocols consists of a series of review-format chapters that provide exten-
sive additional information for those preparing and characterizing gene trans-
v
vi Preface
fer agents. These chapters contain additional information on the use of novel
materials to complex DNA. In a highly detailed series of chapters from the
Middaugh Laboratory at the University of Kansas, a broad range of spectro-
scopic techniques is discussed in the context of characterizing nonviral gene
delivery agents. Finally, a short review on renal gene therapy discusses an
area not well represented elsewhere in treatments of gene therapy. For these
topics, the coverage presented here points out a variety of opportunities for
bringing new approaches to bear on the development and application of nonviral
vectors in the research lab and, eventually, in the clinic.
I regret to note that during the preparation of this book, Dr. Jean-Michel
H. Vos of the University of North Carolina at Chapel Hill passed away. We
are fortunate to have had him contribute to this volume. His work is being
continued by his colleagues, as well as those who will draw on the Vos lab's
studies in their own research.
As with any project of this nature, it would not have been possible with-
out an incredible amount of effort and patience on the part of each and every
contributor, the Series Editor, Professor John M. Walker, and Craig B. Adams
at Humana Press. Thank you to everyone who has supported this project, and
thank you to the readers whose interest in this volume make the efforts to
produce it all worthwhile.
Mark A. Findeis
Contents
Preface .............................................................................................................v
Contributors.....................................................................................................ix
1 Synthesis of Polyampholyte Comb-Type Copolymers Consisting
of a Poly(L-lysine) Backbone and Hyaluronic Acid Side Chains
for DNA Carrier
Atsushi Maruyama and Yoshiyuki Takei............................................ 1
2 Cationic (cid:95)-Helical Peptides for Gene Delivery into Cells
Takuro Niidome and Haruhiko Aoyagi..............................................11
3 Supramolecular Self-Assembly of Poly(ethylene glycol)-Block-Poly
(L-lysine) Dendrimer with Plasmid DNA
Joon Sig Choi and Jong Sang Park..................................................23
4 Water-Soluble Cationic Methacrylate Polymers for Nonviral Gene
Delivery
Gert W. Bos, Daan J. A. Crommelin, and Wim E. Hennink.............35
5 Stabilization of Polycation–DNA Complexes by Surface Modification
with Hydrophilic Polymers
David Oupicky, Martin L. Read, and Thierry Bettinger...................61
6 Use of Disulfide Cationic Lipids in Plasmid DNA Delivery
Fuxing Tang and Jeffrey A. Hughes..................................................79
7 Interactions of Lipid–Oligonucleotide Conjugates with Low-Density
Lipoprotein
Erik T. Rump, Erik A. L. Biessen, Theo J. C. van Berkel,
and Martin K. Bijsterbosch............................................................89
8 Coupling of Nuclear Localization Signals to Plasmid DNA
Carole Neves, Daniel Scherman, and Pierre Wils.........................105
9 Progress Toward a Synthetic Virus: A Multicomponent System
for Liver-Directed DNA Delivery
Bo-Hua Zhong, George Y. Wu, and Catherine H. Wu....................111
10 Characterization of Polyampholyte Comb-Type Copolymer
DNA Carriers
Yoshiyuki Takei, Atsushi Maruyama, Toshihiro Akaike,
and Nobuhiro Sato........................................................................123
vii
viii Contents
11 Methods for Studying the Formation of Polycation–DNA Complexes
and Properties Useful for Gene Delivery
Martin L. Read, Thierry Bettinger, and David Oupicky.................131
12 Characterization of DNA Condensates by Atomic Force Microscopy
Ye Fang and Jan H. Hoh...................................................................149
13 Rapid and Systematic Transfer and Recovery of Large BACs/PACs
into Mammalian Cells by HAEC Retrofitting
Rona S. Scott and Jean-Michel H. Vos...........................................159
14 Systemic Delivery of Therapeutic Proteins by Intramuscular Injection
of Plasmid DNA
Holly M. Horton and Suezanne E. Parker.......................................175
15 Local Delivery of Therapeutic Proteins by Intratumoral Injection
of Plasmid DNA–Lipid Complexes
Holly M. Horton and Suezanne E. Parker.......................................185
16 Nonviral DNA Delivery from Polymeric Systems
Lonnie D. Shea and David J. Mooney.............................................195
17 Promotion of Duplex and Triplex DNA Formation by Polycation
Comb-Type Copolymers
Hidetaka Torigoe and Atsushi Maruyama......................................209
18 Lyophilization of Nonviral Gene Delivery Systems
S. Dean Allison and Thomas J. Anchordoquy...............................225
19 Ultraviolet Absorption and Circular Dichroism Spectroscopy
of Nonviral Gene Delivery Complexes
Chad S. Braun, Lisa A. Kueltzo, and C. Russell Middaugh........253
20 Characterization of Synthetic Gene Delivery Vectors by Infrared
Spectroscopy
Sirirat Choosakoonkriang, Christopher M. Wiethoff,
Lisa A. Kueltzo, and C. Russell Middaugh.................................285
21 Characterization of Cationic Vector-Based Gene Delivery Vehicles
Using Isothermal Titration and Differential Scanning Calorimetry
Brian A. Lobo, Sheila A. Rogers, Christopher M. Wiethoff,
Sirirat Choosakoonkriang, Susan Bogdanowich-Knipp,
and C. Russell Middaugh.............................................................319
22 Light-Scattering Techniques for Characterization of Synthetic
Gene Therapy Vectors
Christopher M. Wiethoff and C. Russell Middaugh.......................349
23 Renal Gene Therapy
Yeong-Hau H. Lien and Li-Wen Lai.................................................377
Index............................................................................................................393
Contributors
TOSHIHIRO AKAIKE • Department of Biomolecular Engineering, Tokyo Insti-
tute of Technology, Yokohama, Japan
S. DEAN ALLISON • P. R. Pharmaceuticals, Ft. Collins, CO
THOMAS J. ANCHORDOQUY • Department of Pharmaceutical Sciences and
Center for Pharmaceutical Biotechnology, University of Colorado Health
Sciences Center, Denver, CO
HARUHIKO AOYAGI •Department of Applied Chemistry, Faculty of
Engineering, Nagasaki University, Nagasaki, Japan
THIERRY BETTINGER • CRC Institute for Cancer Studies, University of
Birmingham, Birmingham, UK
ERIK A. L. BIESSEN • Division of Biopharmaceutics, Leiden/Amsterdam
Center for Drug Research, Leiden, The Netherlands
MARTIN K. BIJSTERBOSCH • Division of Biopharmaceutics, Leiden/Amsterdam
Center for Drug Research, Leiden, The Netherlands
SUSAN BOGDANOWICH-KNIPP • Department of Pharmaceutical Chemistry,
University of Kansas, KS
GERT W. BOS • Department of Pharmaceutics, Utrecht Institute for
Pharmaceutical Sciences, Faculty of Pharmacy, Utrecht University,
Utrecht; and OctoPlus B. V., Pharmaceutical and Biotechnology Product
Development, Leiden, Netherlands
CHAD S. BRAUN • Department of Pharmaceutical Chemistry, University of
Kansas, Lawrence, KS
JOON SIG CHOI • School of Chemistry and Molecular Engineering, Seoul
National University, Seoul, Korea
SIRIRAT CHOOSAKOONKRIANG • Department of Pharmaceutical Chemistry,
University of Kansas, Lawrence, KS
DAAN J. A. CROMMELIN • Department of Pharmaceutics, Utrecht Institute for
Pharmaceutical Sciences, Faculty of Pharmacy, Utrecht University,
Utrecht; and OctoPlus B. V., Pharmaceutical and Biotechnology Product
Development, Leiden, Netherlands
YE FANG • Science and Technology Division, Biochemistry Core Technology,
Corning, Inc., Corning, NY
ix