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Donor-derived, liver-specific protein expression after bone marrow transplantation
D Denison Jenkins1, Konrad Streetz, Monika Tataria
1Department of Surgery, Stanford University School of Medicine, Stanford, California 94305-5733, USA.
Transplantation
|September 28, 2004
Summary
Bone marrow transplantation (BMT) can deliver functional genes to the liver, but expression is low. Liver injury can induce gene expression from BMT, suggesting potential for treating liver disease.
Area of Science:
- Regenerative Medicine
- Hepatology
- Gene Therapy
Background:
- Bone marrow transplantation (BMT) is explored as a method for gene delivery to the liver.
- The role of bone marrow-derived hepatocytes in liver function is not well-defined.
- The clinical utility of BMT for treating liver disease remains uncertain.
Purpose of the Study:
- To quantify protein expression from bone marrow-derived hepatocytes post-BMT.
- To determine if this expression can be induced by liver injury.
Main Methods:
- Mice received BMT from donors expressing human alpha-1 antitrypsin (hAAT).
- Liver injury was induced using adenoviral transduction (Ad-muPA).
- Serum hAAT levels were measured via ELISA in BMT alone and BMT + Ad-muPA groups.
Main Results:
- Baseline hAAT expression in BMT recipients was very low (<80 ng/mL).
- BMT recipients with induced liver injury (BMT + Ad-muPA) showed sustained hAAT levels (~200 ng/mL).
- These differences in hAAT expression were statistically significant.
Conclusions:
- Detectable and persistent serum protein levels indicate successful liver-specific transgene expression after BMT.
- Gene expression is low but can be increased with liver injury.
- Further strategies are being developed to enhance donor-derived protein expression post-BMT.