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The ENCEL system: a somatic cell protein delivery system
S P Squinto1, J A Madri, S Kennedy
1Alexion Pharmaceuticals, Inc, New Haven, CT 06511, USA.
In Vivo (Athens, Greece)
|November 1, 1994
Summary
Microvascular endothelial cells offer a novel approach for gene therapy, enabling sustained therapeutic protein delivery directly into the bloodstream. This ENCEL system provides a stable, effective dosing method for gene-based treatments.
Area of Science:
- Biotechnology
- Gene Therapy
- Cell Biology
Background:
- Somatic cells are investigated for gene therapy, requiring sustained expression and effective delivery.
- Microvascular endothelial cells present advantages for gene delivery due to direct protein secretion and stable expression.
Purpose of the Study:
- To explore microvascular endothelial cells as a gene delivery vehicle for sustained therapeutic protein production.
- To evaluate the potential of the ENCEL system for effective dosing and systemic delivery in gene therapy.
Main Methods:
- Genetically modifying microvascular capillary endothelial cells (ENCEL system).
- Culturing cells in a three-dimensional collagen gel system to form capillary-like structures.
- Utilizing angiogenic factors to induce organized capillary formation in vitro.
Main Results:
- Genetically modified endothelial cells form stable, capillary-like structures in vitro.
- The ENCEL system allows for stable transplantation and removal.
- This system can provide a large number of cells in a small volume for sustained therapeutic protein delivery.
Conclusions:
- Microvascular endothelial cells are a promising vehicle for gene therapy.
- The ENCEL system offers a unique approach for sustained and effective therapeutic protein dosing.
- Further development aims to commercialize a non-human ENCEL system for patient implantation.