Recombinant adeno-associated virus for muscle directed gene therapy

Fisher, Krishna J.; Jooss, Karin; Alston, James; Yang, Yiping; Haecker, Sarah Ehlen; High, Katherine; Pathak, Ravindra; Raper, Steven E.; Wilson, James M.

doi:10.1038/nm0397-306

Cited by 654Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

Recombinant adeno-associated virus for muscle directed gene therapy

Fisher, Krishna J., Jooss, Karin, Alston, James, Yang, Yiping, Haecker, Sarah Ehlen, High, Katherine, Pathak, Ravindra, Raper, Steven E. +1 more·1997·Nature Medicine

DOI 10.1038/nm0397-306

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Abstract

Although gene transfer with adeno-associated virus (AAV) vectors has typically been low, transduction can be enhanced in the presence of adenovirus gene products through the formation of double stranded, non-integrated AAV genomes. We describe the unexpected finding of high level and stable transgene expression in mice following intramuscular injection of purified recombinant AAV (rAAV). The rAAV genome is efficiently incorporated into nuclei of differentiated muscle fibers where it persists as head-to-tail concatamers. Fluorescent in situ hybridization of muscle tissue suggests single integration sites. Neutralizing antibody against AAV capsid proteins does not prevent readministration of vector. Remarkably, no humoral or cellular immune responses are elicited to the neoantigenic transgene product E. coli beta-galactosidase. The favorable biology of rAAV in muscle-directed gene therapy described in this study expands the potential of this vector for the treatment of inherited and acquired diseases.

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After AAV enters a mammalian cell, its DNA persists long-term in the nucleus as episomes, which are less likely than genomic integration to cause oncogenic transformation.
“Upon transduction of a target mammalian cell, the single-stranded AAV DNA undergoes second strand synthesis and subsequently persists long-term within the host nucleus in the form of monomeric or concatemeric episomes [Afione 1996, Fisher 1997]. Compared with integration into the host genome, episomes are associated with minimal risk of causing oncogenic transformation.”
From Correcting visual loss by genetics and prosthetics (2020) · cites this paper as Fisher 1997

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Authors: Fisher, K. J., Jooss, K., Alston, J., Yang, Y., Haecker, S. E., High, K., Pathak, R., Raper, S. E., & Wilson, J. M.
Affiliations: University of Pennsylvania Health System (US)
Children's Hospital of Philadelphia (US)
The Wistar Institute (US)
ORCID: Jooss, Karin
Yang, Yiping
High, Katherine
Raper, Steven E.
Wilson, James M.
Venue: Nature Medicine
Publisher: Springer Science and Business Media LLC
Published: 1997-03
Volume: 3
Issue: 3
Pages: 306-312
ISSN: 1078-8956 (ISSN-L)
1546-170X
DOI: 10.1038/nm0397-306
OpenAlex: W2007854533
Cited by: 654
References: 34
Citations by year: 2026: 1 · 2025: 5 · 2024: 5 · 2023: 6 · 2022: 3 · 2021: 4 · 2020: 5 · 2019: 8 · 2018: 5 · 2017: 8 · 2016: 2 · 2015: 10 · 2014: 6 · 2013: 15 · 2012: 16
FWCI: 39.90
Type: journal article
Language: en
Open access: closed
Licence: www.springer.com/tdm (tdm)
Locations: Nature Medicine (publishedVersion)
PubMed (publishedVersion)
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 › Neurogenetic and Muscular Disorders Research
Keywords: recombinant adeno-associated virus, intramuscular injection, concatemers, transgene expression, beta-galactosidase
MeSH terms: Dependovirus, Animals, DNA, Recombinant, DNA, Viral, Genetic Vectors, Mice, Transgenic, Genetic Therapy, Gene Transfer Techniques, Muscle, Skeletal, Mice

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Recombinant adeno-associated virus for muscle directed gene therapy

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