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Engineered AAVS for CNS Transduction and Peripheral Organ De-Targeting across Species after Systemic Delivery

Flytzanis, Nicholas and Goeden, Nick and Pickel, James and Gradinaru, Viviana (2019) Engineered AAVS for CNS Transduction and Peripheral Organ De-Targeting across Species after Systemic Delivery. Molecular Therapy, 27 (4). Art. No. 102. ISSN 1525-0016. https://resolver.caltech.edu/CaltechAUTHORS:20190502-130921368

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Abstract

In recent years, we have witnessed the development and clinical use of gene therapies at an accelerated pace. The option to introduce, silence, or edit genes greatly increases the therapeutic avenues for a variety of diseases, with adeno-associated viruses (AAVs) being the vehicle of choice due to their low immunogenicity, stable expression, and strong clinical safety record. While the route of delivery has traditionally been localized, systemic administration via the blood is an option in cases where direct injection is impractical or widespread cell populations are affected. However, the naturally occurring AAV serotypes have evolved to broadly infect tissue, an undesirable characteristic for targeting therapeutics to distinct cells.


Item Type:Article
Related URLs:
URLURL TypeDescription
https://doi.org/10.1016/j.ymthe.2019.04.004DOIAbstracts
ORCID:
AuthorORCID
Flytzanis, Nicholas0000-0002-7921-9392
Gradinaru, Viviana0000-0001-5868-348X
Additional Information:© 2019 Elsevier Inc.
Issue or Number:4
Record Number:CaltechAUTHORS:20190502-130921368
Persistent URL:https://resolver.caltech.edu/CaltechAUTHORS:20190502-130921368
Official Citation:ASGCT 22nd Annual Meeting Abstracts, Molecular Therapy, Volume 27, Issue 4, Supplement 1, 2019, Pages 1-465, ISSN 1525-0016, https://doi.org/10.1016/j.ymthe.2019.04.004. (http://www.sciencedirect.com/science/article/pii/S1525001619301698)
Usage Policy:No commercial reproduction, distribution, display or performance rights in this work are provided.
ID Code:95179
Collection:CaltechAUTHORS
Deposited By: Tony Diaz
Deposited On:02 May 2019 20:13
Last Modified:03 Oct 2019 21:11

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