Title:
疾病の薬理シャペロン治療に対する応答性を予測する方法
Document Type and Number:
Japanese Patent JP7277493
Kind Code:
B2
Abstract:
A method of compiling a treatment reference table that indicates the responsiveness of one or more specific mutations of α-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 α-Gal A and compiling a table listing responsive and non-responsive mutant forms of α-Gal A. The specific pharmacological chaperone is 1-deoxygalactonojirimycin or a pharmaceutically salt or ester and the first cell is HEK-293 MSR cell.
Inventors:
Elfrida Benjamin
Buoy Hung Do
Wu Xiao Yang
John Flanagan
Brandon Wustman
Buoy Hung Do
Wu Xiao Yang
John Flanagan
Brandon Wustman
Application Number:
JP2021019383A
Publication Date:
May 19, 2023
Filing Date:
February 09, 2021
Export Citation:
Assignee:
Amicus Therapeutics Incorporated
International Classes:
C12Q1/02; A61K31/445; A61P3/00; C12Q1/34; C12Q1/6869
Foreign References:
| WO2007137072A2 |
Attorney, Agent or Firm:
Sonoda & Kobayashi Patent Attorneys Corporation
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