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Systemic delivery of recombinant proteins by genetically modified myoblasts
1Howard Hughes Medical Institute, University of Michigan Medical Center, Ann Arbor 48109.
Science (New York, N.Y.)
|December 6, 1991
Summary
Genetically engineered myoblasts can deliver therapeutic proteins. These engineered cells stably secreted human growth hormone (hGH) in mice, showing potential for disease treatment.
Area of Science:
- Biotechnology
- Regenerative Medicine
- Molecular Biology
Background:
- Stable systemic delivery of recombinant proteins is crucial for treating various diseases.
- Genetically engineered cells offer a potential platform for sustained protein production and release.
Purpose of the Study:
- To investigate the feasibility of using genetically engineered myoblasts as a system for recombinant protein delivery.
- To assess the in vivo stability and secretion of human growth hormone (hGH) by transfected myoblasts.
Main Methods:
- Stable transfectants of the murine C2C12 myoblast cell line were generated to synthesize and secrete hGH.
- Mice were injected with hGH-transfected myoblasts, and hGH levels in muscle and serum were measured.
- Histological analysis was performed on muscles injected with beta-galactosidase-expressing myoblasts.
Main Results:
- Transfected myoblasts successfully synthesized and secreted high levels of hGH in vitro.
- Mice injected with hGH-transfected myoblasts exhibited significant and stable hGH levels in muscle and serum for at least 3 weeks.
- Injected myoblasts fused with existing muscle fibers to form multinucleated myotubes, indicating successful integration.
Conclusions:
- Genetically engineered myoblasts serve as an effective system for the stable delivery of recombinant proteins into systemic circulation.
- This approach holds promise for developing novel therapeutic strategies for acquired and inherited diseases requiring protein replacement therapy.