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Engineered AAVs for Enhanced Transduction of Submucosal Cells within the Lung Following Intravenous Delivery

Goeden, Nick and Flytzanis, Nicholas and Gradinaru, Viviana (2019) Engineered AAVs for Enhanced Transduction of Submucosal Cells within the Lung Following Intravenous Delivery. Molecular Therapy, 27 (4). Art. No. 101. ISSN 1525-0016. https://resolver.caltech.edu/CaltechAUTHORS:20190502-130505856

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Abstract

Lung diseases are one of the leading causes of morbidity and mortality worldwide, highlighting the need for vectors capable of efficiently transducing cells within the lung. Recent developments in gene delivery have demonstrated that the use of gene therapies to treat disease is a realistic and achievable goal. Adeno-associated viruses (AAVs) have quickly become the vector of choice in both laboratory and clinical settings due to their strong safety record and stable expression in vivo, especially when compared to other viruses. However, the naturally occurring AAVs tend to have severely limited tropisms in lung tissues, as expression following intranasal delivery is generally inefficient and restricted to the airway epithelium. One solution to this obstacle is to utilize a systemically injected AAV in conjunction with inhalant based delivery to cover a broad population of cells throughout the lung.


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:20190502-130505856
Persistent URL:https://resolver.caltech.edu/CaltechAUTHORS:20190502-130505856
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:95178
Collection:CaltechAUTHORS
Deposited By: Tony Diaz
Deposited On:02 May 2019 20:13
Last Modified:03 Oct 2019 21:11

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