Transkaryotic Therapies, Inc.

Shire Human Genetic Therapies, Inc.

Melanie D Williams

Douglas A Treco

Richard F Selden

Marianne Borowski

Carol M Kinoshita

Frances P Gillispie

Peter Francis Daniel

Thomas J Schuetz

Frances P Gillespie

DNA molecules comprising a sequence encoding the signal peptide of hGH linked to a heterologous protein and related cells, methods and fusion proteins.

Nucleic acid encoding a chimeric polypeptide

A therapeutic method whereby an individual suspected of having an &agr;-galactosidase A deficiency, such as Fabry disease, is treated either with (1) human cells that have been genetically modified to overexpress and secrete human &agr;-gal A, or (2) purified human &agr;-gal A obtained from cultured, genetically modified human cells.

The invention provides highly purified &agr;-Gal A, and various methods for purifying it; &agr;-Gal A preparations with altered charge and methods for making those preparations; &agr;-Gal A preparations that have an extended circulating half-life in a mammalian host, and methods for making same; and methods and dosages for administering an &agr;-Gal A preparation to a subject.

Treatment of a &agr;-galactosidase a deficiency

A therepeutic method whereby an individual suspected of having an &agr;-galactosidase A deficiency, such as Fabry disease, is treated either with (1) human cells that have been genetically modified to overexpress and secrete human &agr;-gal A, or (2) purified human &agr;-gal A obtained from cultured, genetically modified human cells.

Therapy for &agr;-galactosidase a deficiency

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.

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Natick, MA, United States of America

Melanie D Williams