Bethann Ann Friedman

US Patent:

55498922, Aug 27, 1996

Filed:

Jun 21, 1993

Appl. No.:

8/080855

Inventors:

BethAnn Friedman - Arlington MA
Michael Hayes - Acton MA

Assignee:

Genzyme Corporation - Cambridge MA

International Classification:

A61K 3847
C12N 942
C12N 1556

US Classification:

424 9461

Abstract:

A pharmaceutical composition comprising remodelled recombinant glucocerebrosidase (GCR) is described that provides a therapeutic effect at doses that are lower then those required using remodelled naturally occurring GCR. A method of treating patients with Gaucher's disease using remodelled recombinant GCR is also provided. In vivo uptake of exogenous molecules can be determined by extracting a mixture of cells from a subject, enriching the target cells in vitro, lysing the cells and determining the amount of exogenous molecules.