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Developing AAV Vectors for More Efficient and Selective Gene Expression in Specific Cell Types of the Nervous System Following Systemic Delivery

Ravindra Kumar, Sripriya and Chan, Ken and Jang, Min J. and Huang, Qin and Brown, David and Dobreva, Tatyana and Kim, Hyun M. and Luo, Yicheng and Hurt, Robert C. and Chen, Xinhong and Deverman, Benjamin E. and Gradinaru, Viviana (2018) Developing AAV Vectors for More Efficient and Selective Gene Expression in Specific Cell Types of the Nervous System Following Systemic Delivery. Molecular Therapy, 26 (5). p. 321. ISSN 1525-0016. doi:10.1016/j.ymthe.2018.05.001.

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Recombinant adeno-associated viruses (rAAVs) are commonly used as gene delivery vehicles in biomedical research and have shown great potential for gene therapy. Systemic administration of AAVs can be used to achieve broad vector distribution. However, key challenges remain with systemic administration of AAVs as their lack of organ and/or cell-type specificity may cofound experiments and cause off-target effects. We and others have evolved AAVs to direct their tropism towards specific organs, such as the brain, after systemic delivery. We previously described CREATE, a Cre-based selection method, and used it to develop AAVs that efficiently transduce the central nervous system (CNS) in adult rodents, namely AAV-PHP.B (Deverman et al, Nat. Biotech., 2016), a further enhanced variant, AAV-PHP.eB (Chan et al., Nat. Neurosci., 2017), and AAV-PHP.S (Chan et al., Nat. Neurosci., 2017), a variant that can efficiently transduce the peripheral nervous system (PNS) and several visceral organs.

Item Type:Article
Related URLs:
URLURL TypeDescription
Ravindra Kumar, Sripriya0000-0001-6033-7631
Chan, Ken0000-0002-8853-5186
Jang, Min J.0000-0002-1536-7177
Dobreva, Tatyana0000-0002-2625-8873
Luo, Yicheng0000-0003-3704-2389
Hurt, Robert C.0000-0002-4347-6901
Deverman, Benjamin E.0000-0002-6223-9303
Gradinaru, Viviana0000-0001-5868-348X
Additional Information:© 2018 American Society of Gene & Cell Therapy. Available online 9 May 2018.
Issue or Number:5
Record Number:CaltechAUTHORS:20180706-160530988
Persistent URL:
Official Citation:ASGCT 21st Annual Meeting Abstracts, Molecular Therapy, Volume 26, Issue 5, Supplement 1, 2018, Pages 1-459, ISSN 1525-0016, (
Usage Policy:No commercial reproduction, distribution, display or performance rights in this work are provided.
ID Code:87626
Deposited By: Tony Diaz
Deposited On:06 Jul 2018 23:13
Last Modified:15 Nov 2021 20:50

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