Описание: Part I Introduction
1. Introduction to Viral Vectors and Other Delivery Methods for Gene Therapy of the Nervous System
Frederic P. Manfredsson
Part II Expression Cassettes
2. Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors
Matthew L. Hirsch, Sonya J. Wolfe, and R. J. Samulski
3. Expression of Multiple Functional RNAs or Proteins from One Viral Vector
Tomas Bjцrklund
4. Regulated Gene Therapy
Ludivine Breger, Erika Elgstrand Wettergren, Luis Quintino, and Cecilia Lundberg
5. Design of shRNA and miRNA for Delivery to the CNS
Gabriela Toro and Christian Mueller
6. Tissue Specific Promoters in the CNS
Sebastian Kьgler
Part III Viral Vector Production
7. Small Scale Recombinant Adeno-Associated Virus Purification
Corinna Burger and Kevin Nash
8. Lentivirus Production and Purification
Matthew J. Benskey and Frederic P. Manfredsson
9. Viral Vector Production: Adenovirus
Julius W. Kim, Ramin A. Morshed, J. Robert Kane, Brenda Auffinger, Jian Qiao, and Maciej S. Lesniak
Part IV Viral Vector Tropism
10. Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids
Michael J. Castle, Heikki T. Turunen, Luk H. Vandenberghe, and John H. Wolfe
11. Altering Tropism of rAAV by Directed Evolution
Damien Marsic and Sergei Zolotukhin
12. Altering Entry Site Preference of Lentiviral Vectors into Neuronal Cells by Pseudotyping with Envelope Glycoproteins
Kenta Kobayashi, Shigeki Kato, Ken0ichi Inoue, Masahiko Takada, and Kazuto Kobayashi
13. Directed Evolution of Adenoviruses
Jason G. Smith
Part V Delivery Methods
14. Intraparenchymal Stereotaxic Delivery of rAAV and Special Considerations in Vector Handling
Matthew J. Benskey and Frederic P. Manfredsson
15. MRI-Guided Delivery of Viral Vectors
Ernesto A. Salegio, John Bringas, Krystof S. Bankiewicz
16. Systemic Gene Therapy for Targeting the CNS
Sara E. Gombash and Kevin D. Foust
17. Widespread Neuronal Transduction of the Rodent CNS via Neonatal Viral Injection
Ji-Yoen Kim, Stacy D. Grunke, and Joanna L. Jankowsky
18. AAV-Mediated Gene Transfer to Dorsal Root Ganglion
Hongwei Yu, Gregory Fischer, and Quinn H. Hogan
19. Gene Therapy of the Peripheral Nervous System: The Enteric Nervous System
Matthew J. Benskey and Frederic P. Manfredsson
20. Gene Therapy of the Peripheral Nervous System: Celiac Ganglia
21. Convection Enhanced Delivery of Recombinant Adeno-Associated Virus into the Mouse Brain
Kevin R. Nash and Marcia N. Gordon
22. Non-Viral Gene Therapy of the Nervous System: Electroporation
XueFeng Ding and Ming Fan
23. Non-Viral, Lipid-Mediated DNA and mRNA Gene Therapy of the Central Nervous System (CNS): Chemical-Based Transfection
James G. Hecker
24. Ex Vivo Gene Therapy Using Human Mesenchymal Stem Cells to Deliver Growth Factors in the Skeletal Muscle of a Familial ALS Rat Model
Masatoshi Suzuki and Clive N. Svendsen
Part VI Gene-Therapy Based Modeling of Neurodegenerative Disorders
25. Gene Therapy Models of Alzheimer's Disease and Other Dementias
Benjamin Combs, Andrew Kneynsberg, and Nicholas M. Kanaan
26. Viral Vector-Based Modeling of Neurodegenerative Disorders: Parkinson's Disease
D. Luke Fischer, Sara E. Gombash, Christopher J. Kemp, Frederic P. Manfredsson, Nicole K. Polinski, Megan F. Duffy, and Caryl E. Sortwell
27. Gene Therapy-Based Modeling of Neurodegenerative Disorders: Huntington's Disease
Deborah Young
Part VII Gene-Therapy for the Treatment of Neurological Disorders
28. Gen
