Viral Vectors for Treating Diseases of the Nervous System

by Latchman

• ISBN13: 9780123668561
• ISBN10: 0123668565
• Edition: 1st
• Format: Hardcover
• Copyright: 2003-08-22
• Publisher: Elsevier Science

Summary

Gene therapy offers considerable potential for the treatment of various incurable diseases of the nervous system. This volume describes a number of different viral vectors developed for achieving high efficiency gene delivery to the brain. Vectors described include those based on adenovirus, adeno-associated virus, Herpes Simplex Virus, lentivirus, and other retroviruses. It also discusses the potential application of such viruses in treating brain tumors, Parkinson's disease, and other diseases of the nervous system. * Provides up-to-date account of gene therapy approaches for incurable neurological disorders * Describes a range of gene delivery methods based on different viruses

Table of Contents

CONTRIBUTORS	ix
SECTION I VIRUS VECTORS FOR USE IN THE CENTRAL NERVOUS SYSTEM
Nonneurotropic Adenovirus: A Vector for Gene Transfer to the Brain and Gene Therapy of Neurological Disorders
PEDRO R. LOWENSTEIN, DONATA SUWELACK, JINWEI HU, XIANPENG YU, MAXIMILIANO JIMENEZ-OALMARONI, SHYAM GOVERDHANA, AND MARIA G. CASTRO
I. Introduction: Some History and Other Vectors	3	(4)
II. The Vector System	7	(2)
III. Developments in the Use of Adenovirus Recombinants as Vectors	9	(30)
IV. Adenoviral Recombinant Vectors: Applications to Basic Neuroscience	39	(5)
V. Adenoviral Recombinant Vectors: Applications to Neurological Gene Therapy	44	(2)
VI. Immune Responses to Recombinant Adenoviral Vectors Delivered into the Brain	46	(2)
VII. Summary: Targeting the Brain with Adenovirus-Derived Vectors	48	(4)
References	52	(13)
Adeno-Associated Viral Vectors
E. LEHTONEN AND L. TENENBAUM
I. Biology of Adeno Associated Virus and Its Recombinant Vectors	65	(9)
II. AAV Vector Production	74	(3)
III. Recombinant AAV-Mediated Transduction in the CNS	77	(5)
IV. Factors Limiting Efficient Transduction	82	(4)
V. New Vectors	86	(1)
VI. Regulation of Transgene Expression	87	(1)
VII. Biosafety	88	(2)
References	90	(9)
Problems in the Use of Herpes Simplex Virus as a Vector
LAWRENCE T. FELDMAN
I. Introduction	99	(1)
II. General Aspects of Lytic Cycle Replication and Latent Infections of Neurons in Vivo	100	(2)
III. Reducing Cytotoxicity of the Virus: The Gene Deletion Approach	102	(1)
IV. Reducing Cytotoxicity of the Virus: The Amplicon Approach	103	(1)
V. The Problem of Long-Term Expression during HSV Latency	104	(2)
References	106	(5)
Lentiviral Vectors
JOHAN JAKOBSSON, CECILIA ERICSON, NINA ROSENQVIST, AND CECILIA LUNDBERG
I. Introduction	111	(1)
II. Lentiviral Vectors	111	(3)
III. Direct Injection of Lentiviral Vectors	114	(1)
IV. Ex Vivo Gene Transfer Using Lentiviral Vectors	115	(3)
V. Concluding Remarks	118	(1)
References	119	(4)
Retroviral Vectors for Gene Delivery to Neural Precursor Cells
RYOICHIRO KAGEYAMA, HIROMI HIRATA, AND JUN HATAKEYAMA
I. Introduction	123	(1)
II. Overview of Retrovirus	124	(6)
III. Generation of Recombinant Retroviral Vectors	130	(8)
IV. Gene Delivery to the Nervous System by Retroviral Vectors	138	(7)
References	145	(6)
SECTION II GENE THERAPY WITH VIRUS VECTORS FOR SPECIFIC DISEASES OF THE NERVOUS SYSTEM
The Principles of Molecular Therapies for Glioblastoma
GEORGE KARPATI AND JOSEPHINE NALBANTOGLU
I. Introduction	151	(1)
II. Review	151	(7)
References	158	(7)
Oncolytic Herpes Simplex Virus for Tumor Therapy
JENNIFER C.C. HU AND ROBERT S. COFFIN
I. Introduction	165	(2)
II. Advantages of Oncolytic Cancer Therapy	167	(1)
III. Advantages of HSV as on Oncolytic Agent	167	(1)
IV. Attenuation of HSV to Provide Tumor-Selective Replication	168	(3)
V. Effects of Preexisting Immunity to HSV	171	(1)
VI. Effects of the Immune Response in Enhancing Tumor Therapy	171	(1)
VII. Clinical Experience with Oncolytic HSV	172	(1)
VIII. Improving Oncolytic HSV	173	(4)
IX. Conclusions	177	(1)
References	178	(7)
Recombinant Retrovirus Vectors for Treatment of Malignant Brain Tumors
NIKOLAI G. RAINOV AND CHRISTOF M. KRAMM
I. Introduction	185	(1)
II. Properties of Retrovirus Vectors and Virus Producer Cells	186	(3)
III. RV-Mediated Brain Tumor Therapy in Animal Models	189	(2)
IV. Clinical Studies with RV Vectors	191	(6)
IX. Conclusions	197	(1)
References	198	(7)
Adeno-Associated Viral Vectors for Parkinson's Disease
SHIN-ICHI MURAMATSU, LIJUN WANG, KÜNIHIKO IKEGUCHI, KEN-ICHT FUJIMOTO, IMAHARU NAKANO, TAKASHI OKADA, HIROAKI MIZUKAMI, YUTAKA HANAZONO, AKIHIRO KUME, IMAHARU NAKANO, AND KEIYA OZAWA
I. Introduction	205	(1)
II. Recombinant Adeno Associated Viral Vector	206	(1)
III Restoration of DA Synthesis in the Striatum	207	(6)
IV. Neuroprotection by GDNF Gene Delivery	213	(3)
V. Conclusions	216	(2)
References	218	(5)
Herpes Simplex Virus Vectors for Parkinson's Disease
DAVID S. LATCHMAN
I. Introduction	223	(1)
II. Gene Therapy and Parkinson's Disease	224	(1)
III. Therapeutic Genes for Parkinson's Disease	224	(2)
IV. Genetics of Parkinson's Disease	226	(2)
V. Gene Delivery to the Nervous System	228	(1)
VI. HSV as a Vector	228	(7)
VII. HSV as a Vector for Parkinson's Disease	235	(2)
VIII. Conclusion	237	(1)
References	238	(5)
Gene Therapy for Stroke
K. ABE AND W.R. ZHANG
I. Introduction	243	(1)
II. Neurotrophic Factors	244	(5)
III. Application of Neurotrophic Factors	249	(4)
IV. Gene Therapy	253	(6)
V. Stem Cells	259	(4)
References	263	(8)
Gene Therapy for Mucopolysaccharidosis
ASSUMPCIO BOSCH AND JEAN MICHEL HEARD
I. Definition and General Considerations on Lysosomal Storage Diseases	271	(2)
II. Addressing Lysosomal Enzymes to the Lysosomes: Enzyme Replacement Therapy	273	(4)
III. Mucopolysaccharidosis	277	(1)
IV. Animal Models for Mucopolysaccharidosis	278	(1)
V. Treatment Strategies for Mucopolysaccharidosis	279	(9)
VI. Perspectives	288	(1)
References	289	(8)
INDEX	297	(22)
CONTENTS OF RECENT VOLUMES	319