Summary of Invention
This invention customizes an erythroid driven lentiviral vector for the long-term and sustained stable expression of high levels of human recombinant protein therapeutics in animal blood. Bone marrow cells are transduced ex vivo with the vector and transplanted into a conditioned recipient animal. The recipient animal is treated with multiple rounds of in vivo drug selection resulting in engraftment and expansion of vector-integrated blood stem cells. The development and maturation of these vector-integrated stem cells triggers the production of high levels of recombinant protein in mature blood cells. The biologically active recombinant protein can be purified from blood cells or, if secreted, from plasma.
- Bone marrow transplantation is less costly and more efficient than current whole animal transgenic cloning saving both time and money during the production of recombinant pharmaceuticals.
- Both male and female animals at any age can be used to harvest recombinant therapeutic proteins utilizing this method leading to an increase in production capacity over harvesting from transgenic animal milk or egg.
- Reduced endogenous protein content in blood and plasma compared to milk or egg allows easier downstream.
- High yield expression (1.7g/L in mice) of biologically active recombinant protein iscomparable to levels produced in transgenic animal milk or egg.
Areas of Application
- Recombinant protein production and purification in animals
- Therapeutic applications
Stage of Development
In vivo proof of concept completed
Michel Sadelain, MD, PhD
Chang AH, et al. (2008) Molecular Therapy. 16:1745-52
U.S patent application published: US2009/0156534
Yashodhara Dash, MBBS, PhD, MBA, CLP
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