Nucleic acid encoding a chimeric polypeptide
AssigneeTranskaryotic Therapies Inc., a Massachusetts corporation
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 α-galactosidase A deficiency, such as Fabry disease, is treated either with (1) human cells that have been genetically modified to overexpress and secrete human α-gal A, or (2) purified human α-gal A obtained from cultured, genetically modified human cells.
Claims35
A method of treatment comprising identifying a patient suspected of having an α-galactosidase A deficiency, and introducing into the patient a human cell genetically modified to overexpress and secrete human α-gal A.
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 α-gal A.
A cultured human cell containing a DNA molecule that (a) encodes a polypeptide comprising human α-gal A linked to an heterologous signal peptide, and (b) permits expression of the polypeptide in the cell.
A clonal cell strain consisting essentially of a plurality of cultured human cells transfected with a DNA molecule that (a) encodes a polypeptide comprising human α-gal A linked to an heterologous signal peptide, and (b) permits overexpression of the polypeptide in the cells.
A clonal cell line consisting essentially of a plurality of immortalized human cells transfected with a DNA molecule that (a) encodes a polypeptide comprising human α-gal A linked to an heterologous signal peptide, and (b) permits overexpression 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 α-gal A.
A process for purifying human α-gal A from a sample, comprising a first chromatography step wherein the sample is passed over a hydrophobic interaction resin.
A therapeutic composition comprising pharmaceutically acceptable purified human α-gal A having N-linked mannose-6-phosphate in a pharmaceutically acceptable carrier.
A DNA molecule encoding a protein comprising the signal peptide of hGH (SEQ ID NO:21) linked to a polypeptide other than hGH.
A cultured human cell containing a DNA molecule that (a) encodes a polypeptide comprising human α-gal A, and (b) permits overexpression of the polypeptide in the cell.
A process for making glycosylated human α-gal A, comprising culturing a human cell containing a DNA molecule that encodes a polypeptide comprising human α-gal A under conditions permitting expression of human α-gal A from said DNA molecule.
A process for purifying human α-gal A from a sample comprising passing said sample over a hydrophobic interaction resin comprising a butyl group as a functional moiety.
Dependencies are inferred from the claim text.