UMMS Affiliation

Department of Neurology

Publication Date


Document Type



Genetics and Genomics | Molecular, Cellular, and Tissue Engineering | Nervous System | Therapeutics | Viruses


Adeno-associated virus (AAV) capsid libraries have generated improved transgene delivery vectors. We designed an AAV library construct, iTransduce, that combines a peptide library on the AAV9 capsid with a Cre cassette to enable sensitive detection of transgene expression. After only two selection rounds of the library delivered intravenously in transgenic mice carrying a Cre-inducible fluorescent protein, we flow sorted fluorescent cells from brain, and DNA sequencing revealed two dominant capsids. One of the capsids, termed AAV-F, mediated transgene expression in the brain cortex more than 65-fold (astrocytes) and 171-fold (neurons) higher than the parental AAV9. High transduction efficiency was sex-independent and sustained in two mouse strains (C57BL/6 and BALB/c), making it a highly useful capsid for CNS transduction of mice. Future work in large animal models will test the translation potential of AAV-F.


AAV capsid library, AAV vector, adeno-associated virus vector, central nervous system, gene delivery, gene therapy, transduction efficiency

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Copyright 2019 The Author(s). This is an open access article under the CC BY-NC-ND license (

DOI of Published Version



Mol Ther Methods Clin Dev. 2019 Oct 23;15:320-332. doi: 10.1016/j.omtm.2019.10.007. eCollection 2019 Dec 13. Link to article on publisher's site

Journal/Book/Conference Title

Molecular therapy. Methods and clinical development


Full author list omitted for brevity. For the full list of authors, see article.

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Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.