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Transgenic pigs produce functional human factor VIII in milk
R K Paleyanda1, W H Velander, T K Lee
1Plasma Derivatives Department Holland Laboratory, Rockville, MD 20855, USA.
Nature Biotechnology
|October 23, 1997
Summary
Researchers developed transgenic pigs to produce recombinant factor VIII (rFVIII) in milk. This novel production method offers a potentially more accessible and cost-effective source for hemophilia A treatment.
Area of Science:
- Biotechnology
- Genetics
- Hematology
Background:
- Hemophilia A is a bleeding disorder caused by factor VIII (FVIII) deficiency.
- Current treatments utilize plasma-derived or recombinant FVIII (rFVIII), which are costly.
- High costs of FVIII therapies limit widespread prophylaxis for hemophilia patients.
Purpose of the Study:
- To establish a novel production system for recombinant factor VIII (rFVIII).
- To express human FVIII cDNA in the mammary glands of transgenic pigs.
- To assess the yield and biological activity of rFVIII produced in porcine milk.
Main Methods:
- Generation of transgenic pigs carrying the human FVIII cDNA under the control of the mouse whey acidic protein gene promoter.
- Collection and analysis of milk from transgenic pigs.
- Characterization of secreted rFVIII using antibody detection, thrombin digestion, and clotting activity assays.
Main Results:
- Successfully generated transgenic pigs expressing human FVIII in their mammary glands.
- Quantified rFVIII secretion in milk at levels up to 2.7 micrograms/ml, significantly higher than plasma levels.
- Confirmed the identity, processing, and biological activity of the secreted rFVIII, which restored normal clotting function in deficient plasma.
Conclusions:
- Mammary gland expression in transgenic pigs provides a high-yield source of biologically active rFVIII.
- This approach represents a promising strategy for reducing the cost of hemophilia A treatment.
- Transgenic animal biotechnology offers a viable alternative for producing therapeutic proteins like rFVIII.