High-accuracy biodistribution analysis of adeno-associated virus variants by double barcode sequencing

Molecular Therapy. Methods & Clinical Development
Damien MarsicSergei Zolotukhin

Abstract

Biodistribution analysis is a key step in the evaluation of adeno-associated virus (AAV) capsid variants, whether natural isolates or produced by rational design or directed evolution. Indeed, when screening candidate vectors, accurate knowledge about which tissues are infected and how efficiently is essential. We describe the design, validation, and application of a new vector, pTR-UF50-BC, encoding a bioluminescent protein, a fluorescent protein and a DNA barcode, which can be used to visualize localization of transduction at the organism, organ, tissue, or cellular levels. In addition, by linking capsid variants to different barcoded versions of the vector and amplifying the barcode region from various tissue samples using barcoded primers, biodistribution of viral genomes can be analyzed with high accuracy and efficiency.

References

Oct 3, 2002·Nature Reviews. Molecular Cell Biology·Gary Thomas
Apr 19, 2005·Nature Biotechnology·Jianmin FangKarin Jooss
Jul 6, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Christophe M DerooseVeerle Baekelandt
Jan 8, 2008·Proceedings of the National Academy of Sciences of the United States of America·Oleg S GorbatyukNicholas Muzyczka
May 6, 2008·Nature Methods·Nathan C ShanerRoger Y Tsien
Jun 2, 2015·Current Pharmaceutical Design·Yarong LiuPin Wang

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Citations

Aug 25, 2017·Human Gene Therapy·Dirk Grimm, Hildegard Büning
Dec 19, 2016·Frontiers in Neuroscience·Shreyasi ChoudhuryShannon E Boye
Dec 11, 2019·Proceedings of the National Academy of Sciences of the United States of America·Marcus DavidssonTomas Björklund
Aug 2, 2017·Virus Genes·Jonas Weinmann, Dirk Grimm
May 1, 2021·Scientific Reports·Elizabeth A CoreyKirill Ukhanov
Jul 24, 2021·Molecular Therapy : the Journal of the American Society of Gene Therapy·Oleksandr KondratovSergei Zolotukhin
Jun 4, 2020·Molecular Therapy. Methods & Clinical Development·Marti Cabanes-CreusLeszek Lisowski

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Datasets Mentioned

BETA
KF926476

Methods Mentioned

BETA
PCR
Illumina sequencing
reverse transcription PCR
Barcode-Seq
electrophoresis
PCRs

Software Mentioned

AAV Barcode - Seq
Python script
DNA
IonTorrent
- barcode
Python

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