Abstract: 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.
Type:
Application
Filed:
December 12, 2002
Publication date:
August 14, 2003
Applicant:
Transkaryotic Therapies Inc., a Massachusetts corporation
Inventors:
Richard F. Selden, Marianne Borowski, Frances P. Gillispie, Carol M. Kinoshita, Douglas A. Treco, Melanie D. Williams