Stem Cell Related Patent Number US6048729

In vivo protein production and delivery system for gene therapy

Selden, Richard F; Wellesley, MA, USA
Treco, Douglas; Arlington, MA, USA
Heartlein, Michael W.; Boxborough, MA, USA

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.

Application Number: US1994000334797

Date Filed: 11/04/94

Dated Published: 04/11/00

Assignee: Transkaryotic Therapies, Inc., Cambridge, MA, USA