Tranfected human cells expressing human .alpha.-galactosidase A protein
AssigneeTranskaryotic Therapies, Inc.
InventorsRichard F. Selden, Marianne Borowski, Frances P. Gillispie, Carol M. Kinoshita, Douglas A. Treco, Melanie D. Williams
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Abstract
A therepeutic method whereby an individual suspected of having an .alpha.-galactosidase A deficiency, such as Fabry disease, is treated either with (1) human cells that have been genetically modified to overexpress and secrete human .alpha.-gal A, or (2) purified human .alpha.-gal A obtained from cultured, genetically modified human cells.
Claims37
A DNA molecule comprising
- a first sequence encoding the hGH signal peptide (SEQ ID NO:21), said first sequence comprising an intron;
- and,
- linked to the 3' end of the first sequence, a second sequence encoding a polypeptide comprising human .alpha.-gal A.
A cultured human cell containing a DNA molecule that (a) encodes a polypeptide comprising human .alpha.-gal A linked to an heterologous signal peptide, and (b) permits expression of the polypeptide in the cell.
A clonal cell strain of cultured human cells transfected with a DNA molecule that (a) encodes a polypeptide comprising human .alpha.-gal A linked to an heterologous signal peptide, and (b) permits expression of the polypeptide in the cells.
A clonal cell line of immortalized human cells transfected with a DNA molecule that (a) encodes a polypeptide comprising human .alpha.-gal A linked to an heterologous signal peptide, and (b) permits expression of the polypeptide in the cells.
A protein comprising (a) the hGH signal peptide (SEQ ID NO:21) linked by a peptide bond to (b) human .alpha.-gal A.
A method for making glycosylated human .alpha.-gal A, comprising culturing a human cell containing a DNA molecule that encodes a polypeptide comprising human .alpha.-gal A and a heterologous signal peptide under conditions permitting expression of human .alpha.-gal A from said DNA molecule.
Dependencies are inferred from the claim text.