Cited by 612Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

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Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8

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Inagaki, Katsuya, Fuess, Sally, Storm, Theresa A., Gibson, Gregory A., Mctiernan, Charles F., Kay, Mark A., Nakai, Hiroyuki·2006·Molecular Therapy

DOI 10.1016/j.ymthe.2006.03.014

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Abstract

It has been recently shown that recombinant adeno-associated virus serotype 8 (rAAV8) is a robust alternative serotype vector that overcomes many of the limitations of rAAV2 and transduces various tissues efficiently and globally through systemic vector administration. AAV9 is a serotype newly isolated from human tissues, but our knowledge of the biology of rAAV9 in vivo is currently limited. Here, we demonstrate by a series of comprehensive side-by-side experiments with rAAV8 and 9 vectors delivered via different routes or at various doses in mice that rAAV9 vectors share the robustness of rAAV8, i.e., (1) very high liver transduction efficiency irrespective of whether vectors are administered intravascularly or extravascularly and (2) substantial transduction in the heart, skeletal muscle, and pancreas by peripheral vein injection. Importantly, rAAV9 transduced myocardium 5- to 10-fold higher than rAAV8, resulting in over 80% cardiomyocyte transduction following tail vein injection of as low as 1.0 x 10(11) particles per mouse. Thus rAAV9, as well as rAAV8, is a robust vector for gene therapy applications and rAAV9 is superior to rAAV8 specifically for cardiac gene delivery by systemic vector administration.

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Authors: Inagaki, K., Fuess, S., Storm, T. A., Gibson, G. A., Mctiernan, C. F., Kay, M. A., & Nakai, H.
Affiliations: University of Pittsburgh (US)
Stanford Medicine (US)
Corresponding author: Nakai, Hiroyuki
ORCID: Inagaki, Katsuya
Storm, Theresa A.
Gibson, Gregory A.
Mctiernan, Charles F.
Kay, Mark A.
Nakai, Hiroyuki
Venue: Molecular Therapy
Publisher: Elsevier BV
Published: 2006-07
Volume: 14
Issue: 1
Pages: 45-53
ISSN: 1525-0016 (ISSN-L)
DOI: 10.1016/j.ymthe.2006.03.014
OpenAlex: W2065380345
Cited by: 610
References: 25
Citations by year: 2026: 26 · 2025: 19 · 2024: 21 · 2023: 18 · 2022: 25 · 2021: 24 · 2020: 27 · 2019: 38 · 2018: 29 · 2017: 24 · 2016: 33 · 2015: 30 · 2014: 25 · 2013: 39 · 2012: 45
FWCI: 21.39
Type: journal article
Language: en
Open access: hybrid
doi.org
Licence: cc-by-nc-nd
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
creativecommons.org/licenses/by-nc-nd/4.0/ (vor, 3751 d)
Locations: Molecular Therapy (publishedVersion, cc-by-nc-nd)
PubMed (publishedVersion)
Funders: National Heart, Lung, and Blood Institute (HL66948, U01HL066948)
National Institute of Diabetes and Digestive and Kidney Diseases (R21DK068636, DK68636)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
Keywords: AAV9, AAV8, adeno-associated virus vectors, systemic gene delivery, cardiac gene transfer, liver transduction, tail vein injection, skeletal muscle transduction, vector dose, gene therapy vectors
MeSH terms: Dependovirus, Animals, Female, Genetic Vectors, Lac Operon, Liver, Male, Mice, Inbred C57BL, Myocardium, Pancreas, Tissue Distribution, Genetic Therapy, Muscle, Skeletal, Mice

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Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8

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