Gene Therapy for Acute and Acquired Diseases

292 Pages·2001·8.481 MB·English

by  Kenneth L. Brigham M.D. (auth.), Phillip Factor D.O., F.C.C.P. (eds.)

Gene Therapy for Acute
and Acquired Diseases

GENE THERAPY FOR ACUTE
AND ACQUlRED DISEASES
edited by
Phillip Factor, D.D., F.C.C.P.
Pulmonary and Critical Care Medicine
Evanston Northwestern Healthcare
and
Northwestern University Medical School
....
"
SPRINGER SCIENCE+BUSINESS MEDIA, LLC
Library of Congress Cataloging-in-Publication Data
Gene therapy for acute and acquired diseases / edited by Phillip Factor.
p. ;cm.
Includes bibliographical references and index.
ISBN 978-1-4613-5668-4 ISBN 978-1-4615-1667-5 (eBook)
DOI 10.1007/978-1-4615-1667-5
1. Gene therapy. 1. Factor, Phillip, 1959-
[DNLM: 1. Gene Therapy. 2. Acquired Immunodeficiency Syndrome--therapy. 3.
Acute Disease--therapy. 4. Critical Illness--therapy. 5. HIV Infections--therapy. QZ 50
G3212520011
RB155.8 .G463 2001
616'.042--dc21
00-051458
Copyright © 2001 Springer Science+Business Media New York
Originally published by Kluwer Academic Publishers in 2001
Softcover reprint ofthe hardcover lst edition 2001
AH rights reserved. N o part of this pub1ication may be reproduced, stored in a retrieval system
or transmitted in any form or by any means, mechanical, photo-copying, recording, or otherwise,
without the prior written permis sion of the publisher, Springer Science +Business Media, LLC.
Printed on acid-free paper.
CONTENTS
Contents v
Contributors vii
Preface Xl
1 Rationale for the Use of Gene Therapy for Critical Illness
Kenneth L. Brigham, M.D. and Roberto Cruz-Gervis, M.D. 1
2 Gene Delivery in the ICU
Daniel J. Weiss, M.D. 17
3 Non-viral Based Gene Therapy for Pulmonary Disease
Myra Stern, M.D. and Eric Alton, M.D. 35
4 Gene Therapy for Acute Lung Injury
Serpil Erzurum, M.D. and Patricia Lemarchand M.D., Ph.D. 53
5 Gene Therapy for Preservation of Alveolar Endothelial and
Epithelial Function
Hazel Lum, Ph.D. and Phillip Factor, D.O. 65
6 Gene Therapy for Fibroproliferative Phase ARDS
Paul N. Reynolds, M.D. and Mitchell A. Olman, M.D. 85
7 Gene Therapy for Asthma
Marc Mathieu, Ph.D. and Pascal Demoly, M.D., Ph.D. 107
8 Gene Therapy for Infectious Diseases
Gokhan M. Mutlu, M.D. and H. Ari Jaffe, M.D. 127
9 Gene Therapy for HIV and AIDS-Related Opportunistic
Infections
Jay K. Kolls, M.D. 147
10 Gene Therapy for Acute Kidney Diseases
Enyu Imai, M.D. and Yoshitaka Isaka, M.D. 159
11 Gene Therapy Strategies to Augment Contractile Function in
Heart Failure
Michael J. Davidson, M.D. and Walter J. Koch, Ph.D. 181
12 Gene Therapy for Cerebrovascular Disease and Central Nervous
System Trauma
R. Loch Macdonald, M.D., Ph.D. 201
13 Gene Therapy for Applications for the Treatment of Acute
Inflammatory Conditions
C. Oberholzer, M.D, A. Oberholzer, M.D.,
L.L. Moldawer, Ph.D. 225
14 Facilitation of Organ Transplantation with Gene Therapy
Nick Giannoukakis, Ph.D., and Paul D. Robbins, Ph.D. 247
15 Cardiac Angiogenesis and Gene Therapy: A Strategy for
Myocardial Revascularization
Todd K. Rosengart, M.D. 273
Index 285
CONTRIBUTORS
Eric Alton, M.D., FRCP Pascal Demoly, M.D., Ph.D.
Department of Gene Therapy, Service des Maladies
Imperial College at the National Respiratoires-INSERM U454,
Heart and Lung Institute, London, Hopital Arnaud de Villeneuve,
U.K 34295 Montpellier Cedex 5,
[email protected] France
[email protected]
Kenneth L. Brigham, M.D.
Division of Allergy, Pulmonary Serpil Erzurum, M.D.
and Critical Care Medicine, Pulmonary and Critical Care
Vanderbilt University Medical Medicine, and Cancer Biology,
Center Lerner Research Institute,
Nashville, TN 37232 Cleveland Clinic Foundation,
Ken.Brigham@mcmail. Cleveland, Ohio 44195
vanderbilt.edu [email protected]
Roberto Cruz-Gervis, M.D. Phillip Factor, D.O.
Meharry Medical College Section of Pulmonary and Critical
Nashville, TN 37208 and Care Medicine,
Division of Allergy, Pulmonary Evanston Northwestern
and Critical Care Medicine, Healthcare,
Vanderbilt University Medical 2650 Ridge Ave.
Center Evanston, IL 60201
Nashville, TN 37232 [email protected]
Michael J. Davidson, M.D. Nick Giannoukakis, Ph.D.
Department of Surgery, Department of Molecular Genetics
Duke University Medical Center, and Biochemistry, University of
Durham, NC Pittsburgh School of Medicine,
Pittsburgh, PA, 15261
ngiannl [email protected]
Enyu Imai, M.D. Hazel Lum, Ph.D.
Division of Nephrology, Department of Pharmacology
Department of Internal Medicine Rush University
and Therapeutics (A8), Graduate Rush Presbyterian St. Luke's
School of Medicine, Osaka Medical Center
University, 2242 W. Harrison St.; Suite 260
Suite 5650-0871 Chicago, IL 60612
Osaka, Japan [email protected]
[email protected]
R. Loch Macdonald, M.D.,
Yoshitaka Isaka, M.D. Ph.D.
Division of Nephrology, Section of Neurosurgery,
Department of Internal Medicine MC3026, Department of Surgery,
and Therapeutics, Graduate School University of Chicago Medical
of Medicine, Osaka University, Center and Pritzker School of
Osaka, Japan Medicine,
Chicago, IL 60037
H. Ari Jaffe, M.D. [email protected].
Section of Respiratory and Critical uchicago.edu
Care Medicine, University of
Illinois at Chicago, Chicago, IL Marc Mathieu, Ph.D.
60612 INSERM U454,
[email protected] Hopital Arnaud de Villeneuve,
34295 Montpellier Cedex 5,
Walter J. Koch, Ph.D. France
Department of Surgery,
Duke University Medical Center, Lyle L. Moldawer, Ph.D.
Durham, NC University of Florida
[email protected] College of Medicine,
Gainesville, FL 32610
Jay K. Kolls, M.D. [email protected]
Louisiana State University Health
Sciences Gene Therapy Program, Gijkhan M. Mutlu, M.D.
New Orleans, LA 70112 Pulmonary and Critical Care
[email protected] Medicine, Evanston Northwestern
Healthcare, Evanston, IL 60201
Patricia Lemarchand M.D., [email protected]
Ph.D.
INSERM EOO-16, Andreas Oberholzer, M.D.
Faculte de Medecine Necker University of Florida
Enfants Malades, College of Medicine,
Paris, France Gainesville, FL 32610
[email protected] [email protected]
Caroline V. Oberholzer, M.D. Paul D. Robbins, Ph.D.
University of Florida Department of Molecular Genetics
College of Medicine, and Biochemistry, University of
Gainesville, FL 32610 Pittsburgh School of Medicine,
[email protected] Pittsburgh, PA, 15261
[email protected]
Mitchell A. Olman, M.D.
Division of Pulmonary, Allergy Myra Stern, Ph.D., MRCP
and Critical Care Medicine, Univ. Department of Gene Therapy,
of Alabama at Birmingham Imperial College at the National
Birmingham, AL 35294 Heart and Lung Institute, London,
[email protected] U.K.
[email protected]
Paul N. Reynolds, MBBS
PhD,FRACP Daniel J. Weiss, M.D., Ph.D.
Division of Human Gene Therapy Pulmonary and Critical Care
Department of Medicine Medicine,
University of Alabama at Fred Hutchinson Cancer Research
Birmingham Center and University of
Birmingham, AL 35294 Washington School of Medicine,
[email protected] Seattle, W A 98109
[email protected]
Todd Rosengart
Division of Cardiothoracic
Surgery, Evanston Northwestern
Healthcare,
2650 Ridge Ave.
Evanston, IL 60201
[email protected]
PREFACE
In recent years much enthusiasm and energy has been directed toward the
development of human gene therapies, especially for inherited conditions and
cancers. However, current gene transfer technology is limited in its
transduction efficiency and ability to permanently and safely correct genomic
defects. Thus, the promise of gene therapy for these conditions is as yet
unrealized. The progression of gene transfer technology will eventually
surmount these limitations.
As our understanding of disease processes has grown it has become apparent
that protein loss and/or dysfunction contributes to the pathophysiology of
many acute, acquired medical conditions. Gene therapy offers the possibility
of restoration (or improvement) of protein function via gene overexpression
in these conditions. Gene transfer also offers the potential for gain-of
function via expression of genes not otherwise present in a particular cell or
organ. Most notably, acute conditions do not require permanent genetic
correction. Thus gene transfer represents a highly efficient intracellular
pharmaceutical delivery system that may be better suited for acute, acquired
conditions than heritable conditions. However, like heritable conditions,
current limitations of gene transfer technology have slowed the clinical
application of gene therapy for acute disorders.
Included in this assemblage are selected examples of ongoing studies in
molecular genetics that have the potential to evolve into human therapies for
acute illnesses. The chapters included are intended to highlight lesser known
applications of gene therapy for acquired disorders. It is expected that human
gene therapy trials for these conditions will be forthcoming in the near future
leading to previously unimaginable therapies. Thus this first-ever book about
gene therapy for acute and acquired diseases is intended to serve as a glimpse
into our future.