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METHOD TO PREDICT response to Pharmacological chaperone TREATMENT OF DISEASES
专利权人:
AMICUS THERAPEUTICS; INC.
发明人:
BENJAMIN, ELFRIDA,DO, HUNG, V.,WU, XIAOYANG,FLANAGAN, JOHN,WUSTMAN, BRANDON
申请号:
DK09710178
公开号:
DK2252313T3
申请日:
2009.02.12
申请国别(地区):
DK
年份:
2015
代理人:
摘要:
A method of compiling a treatment reference table that indicates the responsiveness of one or more specific mutations of ±-Gal A to a specific pharmacological chaperone, the method comprising: analysing in vitro the response to the specific pharmacological chaperone in a host cell that has been transformed with a nucleic acid vector that encodes a first mutant ±-Gal A and compiling a table listing responsive and non-responsive mutant forms of ±-Gal A. The specific pharmacological chaperone is 1-deoxygalactonojirimycin or a pharmaceutically salt or ester and the first cell is HEK-293 MSR cell.
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