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Systemic Delivery of Recombinant Proteins by Genetically Modified Myoblasts
Abstract
The ability to stably deliver recombinant proteins to the systemic circulation would facilitate the treatment of a variety of acquired and inherited diseases. To explore the feasibility of the use of genetically engineered myoblasts as a recombinant protein delivery system, stable transfectants of the murine C2C12 myoblast cell line were produced that synthesize and secrete high levels of human growth hormone (hGH) in vitro. Mice injected with hGH-transfected myoblasts had significant levels of hGH in both muscle and serum that were stable for at least 3 weeks after injection. Histological examination of muscles injected with β-galactosidase-expressing C2C12 myoblasts demonstrated that many of the injected cells had fused to form multinucleated myotubes. Thus, genetically engineered myoblasts can be used for the stable delivery of recombinant proteins into the circulation.
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Cited by
Recombinant adeno-associated virus for muscle directed gene therapy
Fisher, K. J., Jooss, K., Alston, J., Yang, Y., Haecker, S. E., High, K., Pathak, R., Raper, S. E., & Wilson, J. M. (1997) · Nature Medicine · doi:10.1038/nm0397-306 · Cited by 654
Metadata
- Authors
- Barr, E., & Leiden, J. M.
- Venue
- Science
- Published
- 1991-12-06
- Volume
- 254
- Issue
- 5037
- Pages
- 1507-1509
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W2034511195
- Cited by
- 281
- References
- 0
- FWCI
- 9.77
- Type
- journal article
- Language
- en
- Open access
- closed
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- Barr, E., & Leiden, J. M. (1991). Systemic Delivery of Recombinant Proteins by Genetically Modified Myoblasts. Science. https://doi.org/10.1126/science.1962212
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
