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High-Resolution Histological Landscape of AAV DNA Distribution in Cellular Compartments and Tissues following Local and Systemic Injection

Authors
  • Zhao, Junling1
  • Yue, Yongping1
  • Patel, Aman1, 2
  • Wasala, Lakmini1
  • Karp, Jacob F.1
  • Zhang, Keqing1
  • Duan, Dongsheng1, 3, 4, 5
  • Lai, Yi1
  • 1 Department of Molecular Microbiology and Immunology, School of Medicine, University of Missouri, Columbia, MO 65212, USA
  • 2 School of Medicine, Saint Louis University, St. Louis, MO 63104, USA
  • 3 Department of Biomedical Sciences, College of Veterinary Medicine, University of Missouri, Columbia, MO 65212, USA
  • 4 Department of Neurology, School of Medicine, University of Missouri, Columbia, MO 65212, USA
  • 5 Department of Bioengineering, University of Missouri, Columbia, MO 65212, USA
Type
Published Article
Journal
Molecular Therapy — Methods & Clinical Development
Publisher
Elsevier
Publication Date
Aug 11, 2020
Volume
18
Pages
856–868
Identifiers
DOI: 10.1016/j.omtm.2020.08.006
PMID: 32953935
PMCID: PMC7479330
Source
PubMed Central
Keywords
License
Unknown

Abstract

Using RNAscope, for the first time, the high-resolution body-wide organ, tissue, and cell biodistribution profile of the AAV9 vector was delineated following intravenous and intramuscular injection in a mouse model of Duchenne muscular dystrophy. These findings add a new dimension to our understanding of AAV transduction biology in mammals.

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