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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Adipose tissue-derived mesenchymal stem cell-based liver gene delivery
Hong Li1, Bin Zhang, Yuanqing Lu
1Departments of Pharmaceutics, University of Florida, Gainesville, FL, USA.
Journal of Hepatology
|December 21, 2010
Summary
Adipose tissue-derived stem cells (AT-MSCs) can be genetically modified and transplanted into the liver to treat alpha-1-antitrypsin deficiency. This approach shows sustained therapeutic protein expression without immune rejection, offering a novel gene therapy for liver disease.
Area of Science:
- Regenerative Medicine
- Gene Therapy
- Stem Cell Biology
Background:
- Adipose tissue-derived mesenchymal stem cells (AT-MSCs) are a viable alternative to bone marrow-derived mesenchymal stem cells (BM-MSCs).
- AT-MSCs offer potential for regenerative medicine due to accessibility and abundance.
Purpose of the Study:
- To investigate the feasibility of using AT-MSCs for liver gene delivery.
- To evaluate AT-MSC-based gene therapy for alpha-1-antitrypsin deficiency.
Main Methods:
- Mouse AT-MSCs were transduced with recombinant adeno-associated viral vectors (rAAV).
- Transduced AT-MSCs were transplanted into mouse livers.
Main Results:
- AT-MSCs successfully expressed the human alpha-1-antitrypsin (hAAT) transgene after rAAV transduction.
- Engrafted AT-MSCs sustained hAAT serum levels in recipients.
- No anti-hAAT antibodies were detected, indicating no immune response.
Conclusions:
- AT-MSCs can be effectively transduced by rAAV vectors for gene therapy applications.
- AT-MSCs engraft in the liver, express therapeutic proteins, and do not elicit an immune response.
- AT-MSC-based gene therapy is a promising approach for treating liver diseases like alpha-1-antitrypsin deficiency.
