Cited by 153Virus-based gene therapy researchRNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

Recombinant Adeno-Associated Viral Vectors Mediate Long-Term Transgene Expression in Muscle

Clark, K. Reed, Sferra, Thomas J., Johnson, Philip R.·1997·Human Gene Therapy

Abstract

Gene transfer to muscle holds overt promise for the treatment of inherited myopathies, lysosomal storage disorders, and serum protein deficiencies. In addition, muscle could provide a reservoir for delivery of therapeutic molecules like blood clotting factors, erythropoietin, or insulin. To date, successful gene transfer to muscle has been limited by the inefficiency of the vector delivery systems and the transient nature of gene expression. In this paper, we show that a vector based on recombinant adeno-associated virus (rAAV) can efficiently transduce adult mouse skeletal muscle. Transduced myofibers escape immune elimination and transgene expression is robust beyond 5 months. Importantly, input vector DNA appears to undergo conversion from single-stranded genomes to high-molecular-weight concatameric forms. These data suggest that rAAV might have a significant advantage over many other viral and nonviral gene delivery methods, and holds significant promise as a vector for gene transfer to mature muscle.

Integrity signals

Registry

Crossref · 10.1089/hum.1997.8.6-659

Cites

62 of 62 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Claims the paper makes

This paper’s full text isn’t openly available, so its claims and self-check weren’t run. The registry and citation checks still apply.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Cites

Flagged first, then by citations

Cited by

Sorted by citations

Authors
Clark, K. R., Sferra, T. J., & Johnson, P. R.
Affiliations
Nationwide Children's Hospital (US)
Corresponding author
Johnson, Philip R.
Venue
Human Gene Therapy
Published
1997-04-10
Volume
8
Issue
6
Pages
659-669
ISSN
  • 1043-0342 (ISSN-L)
  • 1557-7422
OpenAlex
W1979746830
Cited by
153
References
62
Citations by year
2026: 1 · 2024: 1 · 2020: 3 · 2019: 3 · 2017: 2 · 2016: 2 · 2015: 5 · 2014: 3 · 2013: 4
FWCI
14.16
Type
journal article
Language
en
Open access
closed
Locations
  • Human Gene Therapy (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, skeletal muscle transduction, long-term transgene expression, concatemeric DNA, immune evasion, neutralizing antibodies, vector immunogenicity, inherited myopathies, lysosomal storage disease
MeSH terms
Dependovirus, Animals, beta-Galactosidase, DNA, Recombinant, Gene Expression Regulation, HeLa Cells, Humans, Mice, Inbred BALB C, Gene Transfer Techniques, Muscle, Skeletal, Transgenes, Mice

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review