Gene transfer to the CNS using recombinant adeno-associated virus
UMass Chan Affiliations
Department of Microbiology and Physiological SystemsDepartment of Neurology
Gene Therapy Center
Document Type
Book ChapterPublication Date
2013-05-01Keywords
DependovirusGenetic Vectors
Genetic Therapy
Gene Transfer Techniques
Central Nervous System
UMCCTS funding
Biomedical Engineering and Bioengineering
Genetics and Genomics
Microbiology
Metadata
Show full item recordAbstract
Recombinant adeno-associated virus (rAAV) vectors are great tools for gene transfer due to their ability to mediate long-term gene expression. rAAVs have been used successfully as gene transfer vehicles in multiple animal models of CNS disorders, and several clinical trials are currently underway. rAAV vectors have been used at various stages of development with no apparent toxicity. There are multiple ways of delivering AAV vectors to the mouse CNS, depending on the stage of development. In neonates, intravascular injections into the facial vein are often used. In adults, direct injections into target regions of the brain are achieved with great spatiotemporal control through stereotaxic surgeries. Recently, discoveries of new AAV vectors with the ability to cross the blood brain barrier have made it possible to target the adult CNS by intravascular injections. Curr. Protoc. Microbiol. 29:14D.5.1-14D.5.18. (c) 2013 by John Wiley and Sons, Inc.Source
Curr Protoc Microbiol. 2013 May;Chapter 14:Unit14D.5. doi: 10.1002/9780471729259.mc14d05s29. Link to article on publisher's site
DOI
10.1002/9780471729259.mc14d05s29Permanent Link to this Item
http://hdl.handle.net/20.500.14038/30212PubMed ID
23686825Notes
Co-author Seemin Seher Ahmed is a doctoral student in the Interdisciplinary Graduate Program in the Graduate School of Biomedical Sciences (GSBS) at UMass Medical School.
Related Resources
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10.1002/9780471729259.mc14d05s29