Gene therapy for neurological disorders : methods and protocols / edited by Fredric P. Manfredsson.
Contributor(s): Manfredsson, Fredric P [editor.]Material type: TextSeries: Methods in molecular biology (Clifton, N.J.): v. 1382.; Springer protocols (Series): Publisher: New York, NY : Humana Press : Springer, ©2016Description: 1 online resource (xiv, 493 pages) : illustrations (some color)Content type: text Media type: computer Carrier type: online resourceISBN: 9781493932719; 1493932713Subject(s): Nervous system -- Diseases -- Gene therapy -- Laboratory manuals | Nervous System Diseases -- therapy | Genetic Therapy -- methods | Genetic Vectors | Medical -- Genetics | Medical -- Neurology | Medical genetics | Neurology & clinical neurophysiology | Medical -- Neuroscience | NeurosciencesGenre/Form: Electronic books. | Laboratory manuals. Additional physical formats: Print version:: Gene therapy for neurological disorders.DDC classification: 616.8/0442 LOC classification: RC346.4RC321-580Online resources: Click here to access online
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Includes bibliographical references and index.
Introduction to viral vectors and other delivery methods for gene therapy of the nervous system / Fredric P. Manfredsson -- Delivering transgenic DNA exceeding the carrying capacity of AAV vectors / Matthew L. Hirsch, Sonya J. Wolf, and R.J. Samulski -- Expression of multiple functional RNAs or proteins from one viral vector / Tomas Björklund -- Regulated gene therapy / Ludivine Breger [and others] -- Design of shRNA and miRNA for delivery to the CNS / Gabriela Toro Cabrera and Christian Mueller -- Tissue-specific promoters in the CNS / Sebastian Kügler -- Small-scale recombinant adeno-associated virus purification / Corinna Burger and Kevin R. Nash -- Lentivirus production and purification / Matthew J. Benskey and Fredric P. Manfredsson -- Viral vector production : adenovirus / Julius W. Kim [and others] -- Controlling AAV tropism in the nervous system with natural and engineered capsids / Michael J. Castle [and others] -- Altering tropism of rAAV by directed evolution / Damien Marsic and Sergei Zolotukhin -- Altering entry site preference of lentiviral vectors into neuronal cells by pseudotyping with envelope glycoproteins / Kenta Kobayashi [and others] -- Directed evolution of adenoviruses / Jason G. Smith -- Intraparenchymal stereotaxic delivery of rAAV and special considerations in vector handling / Matthew J. Benskey and Fredric P. Manfredsson -- MRI-guided delivery of viral vectors / Ernesto A. Salegio, John Bringas, and Krystof S. Bankiewicz -- Systemic gene therapy for targeting the CNS / Sara E. Gombash and Kevin D. Foust -- Widespread neuronal transduction of the rodent CNS via neonatal viral injection / Ji-Yoen Kim, Stacy D. Grunke, and Joanna L. Jankowsky -- AAV-mediated gene transfer to dorsal root ganglion / Hongwei Yu, Gregory Fischer, and Quinn H. Hogan -- Gene therapy of the peripheral nervous system : the enteric nervous system / Matthew J. Benskey and Fredric P. Manfredsson -- Gene therapy of the peripheral nervous system : celiac ganglia / Bradley Hammond and David L. Kreulen -- Convection enhanced delivery of recombinant adeno-associated virus into the mouse brain / Kevin R. Nash and Marcia N. Gordon -- Nonviral gene therapy of the nervous system : electroporation / Xue-Feng Ding and Ming Fan -- Non-viral, lipid-mediated DNA and mRNA gene therapy of the central nervous system (CNS) : chemical-based transfection / James G. Hecker -- 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 -- Gene therapy models of Alzheimer's disease and other dementias / Benjamin Combs, Andrew Kneynsberg, and Nicholas M. Kanaan -- Viral vector-based modeling of neurodegenerative disorders : Parkinson's disease / D. Luke Fischer [and others] -- Gene therapy-based modeling of neurodegenerative disorders : Huntington's disease / Deborah Young -- Gene therapy for the treatment of neurological disorders : amyotrophic lateral Sslerosis / Zachary T. McEachin, Anthony Donsante, and Nicholas Boulis -- Stereotaxic surgical targeting of the nonhuman primate caudate and putamen : gene therapy for Huntington's disease / Jodi L. McBride and Randall L. Clark -- Gene therapy for the treatment of neurological disorders : metabolic disorders / Dominic J. Gessler and Guangping Gao -- Gene therapy for the treatment of neurological disorders : central nervous system neoplasms / Neha Kamran [and others] -- AAV2-neurturin for Parkinson's disease : what lessons have we learned? / Jeffrey H. Kordower.
Online resource; title from PDF title page (SpringerLink, viewed November 30, 2015).
This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results. Chapters in this book disseminate bits of unknown information that are important to consider during the course of experimentation and will answer questions such as: What delivery vehicle do you use?; How will you ensure that your vector retains stability?; What expression system best fits your needs?; What route will you choose to deliver your gene therapy agent?; How will you model the neurodegenerative disorder that you aim to investigate, and what are the proven methods to treat these disorders in preclinical models? Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and thorough, Gene Therapy for Neurological Disorders: Methods and Protocols, is a compilation of protocols and instructive chapters intended to give researchers, clinicians, and students of all levels, a foundation upon which future gene therapy experiments can be designed.