Understanding Gene Therapy

by Lemoine, Nicholas R.; Vile, Richard G.

• ISBN13: 9780387915128
• ISBN10: 0387915125
• Format: Paperback
• Copyright: 1999-12-01
• Publisher: Taylor & Francis

Summary

(BIOS Scientific Publishers) Imperial College of Medicine, London, UK. Provides an introduction to this expanding field. Discusses what gene therapy is, how genes are delivered, which diseases can be treated, the status of current clinical trials, ethical issues, and future prospects. For students and clinicians. Softcover. DNLM: Gene Therapy.

Table of Contents

Contributors	ix
Abbreviations	xi
Preface	xvii
What is gene therapy?	1	(10)
J. Drew
L.-A. Martin
Introduction	1	(1)
Germline gene therapy	1	(1)
Somatic gene therapy	2	(2)
Viral systems	4	(2)
Nonviral systems	6	(2)
Candidate diseases for application of gene therapy	8	(1)
Summary	9	(1)
References	9	(1)
Further reading	10	(1)
Why gene therapy?	11	(10)
L.-A. Martin
J. Drew
Introduction	11	(1)
What makes a disease suitable for gene therapy?	11	(1)
Cystic fibrosis	12	(1)
Adenosine deaminase deficiency	13	(1)
Familial hypercholesterolemia	13	(1)
Cancer -- could gene therapy provide a cure?	14	(2)
Infectious diseases	16	(1)
Ethical and regulatory considerations	17	(1)
Gene therapy industry -- an explosion waiting to happen?	17	(2)
Future prospects for gene therapy	19	(1)
Further reading	19	(2)
Viral delivery systems for gene therapy	21	(22)
S.J. Murphy
Introduction	21	(1)
Retrovirus-based vectors	22	(4)
Adenovirus-based vectors	26	(7)
Adeno-associated virus-based vectors	33	(1)
Herpes simplex virus-based vectors	34	(1)
Other viral vectors and hybrid vector systems	35	(3)
References	38	(5)
Nonviral delivery systems for gene therapy	43	(28)
A.D. Miller
Introduction	43	(2)
Cationic liposome/micelle-based nonviral delivery systems	45	(7)
Cationic polymer-based nonviral delivery systems	52	(5)
Alternative chemical nonviral delivery systems	57	(1)
Physical nonviral delivery systems	58	(1)
Future prospects for nonviral delivery systems	59	(1)
References	60	(11)
Gene therapy for monogenic diseases	71	(12)
G. Vassaux
Introduction	71	(1)
Gene therapy for adenosine deaminase deficiency	71	(7)
Gene therapy for cystic fibrosis	78	(4)
Conclusion	82	(1)
Further reading	82	(1)
Gene therapy for multifactorial genetic disorders	83	(16)
A.S. Rigg
Introduction	83	(1)
Colorectal cancer	83	(9)
Atherosclerotic vascular diseases	92	(2)
Diabetes mellitus	94	(3)
Summary	97	(1)
Further reading	98	(1)
Gene therapy for infectious diseases	99	(26)
S. Fidler
J. Weber
Introduction	99	(1)
Strategies for antiviral gene therapy	100	(13)
The use of gene therapy to manipulate the immune response	113	(9)
Conclusions	122	(1)
References	122	(3)
Targeting of gene delivery systems	125	(16)
L.-A. Martin
Introduction	125	(1)
Ex vivo gene transfer	125	(2)
Transduction targeting	127	(6)
Liposome vectors	133	(1)
Molecular conjugates	134	(1)
Targeting at the transcriptional level	135	(3)
Conclusion	138	(1)
Further reading	139	(2)
Gene therapy in the clinic: human trials of gene therapy	141	(14)
T. Valere
Introduction	141	(1)
Gene therapy around the globe	141	(2)
Vectors and routes of administration	143	(1)
Which genes for which diseases?	143	(2)
Results: the global picture	145	(7)
Conclusion	152	(1)
References	152	(2)
Further reading	154	(1)
Ethical issues in gene therapy	155	(8)
N.C. Nevin
Introduction	155	(1)
Supervision of gene therapy research	156	(1)
Consent to research	157	(1)
Confidentiality	158	(1)
Medical surveillance of gene therapy patients	159	(1)
Germline gene therapy	159	(1)
In utero gene therapy	160	(1)
Conclusion	161	(1)
References	162	(1)
Prospects for gene therapy	163	(1)
N.R. Lemoine
Introduction	163	(1)
Mechanisms of gene transfer and expression	163	(1)
Development of models of disease and therapy	164	(1)
Cycles of clinical development -- beyond the phase I trial	164	(1)
References	165