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Stem Cell Related Patent Number US6048729
| Title: | In vivo protein production and delivery system for gene therapy | | Inventors: | Selden, Richard F; Wellesley, MA, USA
Treco, Douglas; Arlington, MA, USA
Heartlein, Michael W.; Boxborough, MA, USA | | Summary: | This invention describes novel cells of vertebrate origin, primarily mammalian origin, comprised of exogenous DNA-encoding therapeutic proteins that are useful as a delivery system of products for gene therapy. Described herein are methods by which the primary and secondary somatic cells are transfected with exogenous genetic material which encodes a desired therapeutic product, as well as methods of producing clonal cell strains or heterogenous cell strains, methods of gene therapy in which the transfected primary or secondary cells are used, and methods of producing antibodies using the transfected primary or secondary cells. Further disclosed are properties of the primary and secondary somatic cells such as fibroblasts, keratinocytes, epithelial cells, endothelial cells, glial cells, neural cells, formed elements of the blood, muscle cells, and other somatic cells which can be cultured. Also described are somatic cell precursors, which have been transfected with exogenous DNA which is stably integrated into their genomes or is expressed in the cells episomally. The exogenous DNA either encodes a product, such as a translational product such as a protein, or a transcriptional product such as a ribozyme or an anti-sense nucleic acid sequence, which either produces a therapeutic product or is itself a therapeutic product, such that the DNA binds to a cellular regulatory protein or alters gene expression. | | Abstract: | The present invention relates to transfected primary and secondary somatic cells of vertebrate origin, particularly mammalian origin, transfected with exogenous genetic material (DNA) which encodes a desired (e.g., a therapeutic) product or is itself a desired (e.g., therapeutic) product, methods by which primary and secondary cells are transfected to include exogenous genetic material, methods of producing clonal cell strains or heterogenous cell strains, methods of gene therapy in which the transfected primary or secondary cells are used, and methods of producing antibodies using the transfected primary or secondary cells.
The present invention includes primary and secondary somatic cells, such as fibroblasts, keratinocytes, epithelial cells, endothelial cells, glial cells, neural cells, formed elements of the blood, muscle cells, other somatic cells which can be cultured and somatic cell precursors, which have been transfected with exogenous DNA which is stably integrated into their genomes or is expressed in the cells episomally. The exogenous DNA either encodes a product, such as a translational product (e.g., a protein) or a transcriptional product (e.g., a ribozyme or an anti-sense nucleic acid sequence) which is a therapeutic product or is itself a therapeutic product (e.g., DNA which binds to a cellular regulatory protein or alters gene expression). | | US Patent Website: | Click Here for Full Text of Patent | | Title Number: | US6048729 | | Application Number: | US1994000334797 | | Date Filed: | 04/11/1994 | | Date Published: | 11/04/2000 | | Assignee: | Transkaryotic Therapies, Inc., Cambridge, MA, USA |
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