UMass Chan Medical School Faculty Publications


Gene transfer to the CNS using recombinant adeno-associated virus

UMMS Affiliation

Gene Therapy Center; Department of Neurology; Department of Microbiology and Physiological Systems

Publication Date


Document Type

Book Chapter


Dependovirus; Genetic Vectors; Genetic Therapy; Gene Transfer Techniques; Central Nervous System


Biomedical Engineering and Bioengineering | Genetics and Genomics | Microbiology


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.


UMCCTS funding

DOI of Published Version



Curr Protoc Microbiol. 2013 May;Chapter 14:Unit14D.5. doi: 10.1002/9780471729259.mc14d05s29. Link to article on publisher's site


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

Link to Article in PubMed

Journal/Book/Conference Title

Current protocols in microbiology

PubMed ID