ALMA MATER STUDIORUM - UNIVERSITA DI BOLOGNA;AMICUS THERAPEUTICS, INC.
发明人:
CIANI, ELISABETTA,LACCONE, FRANCO,CLARK, SEAN
申请号:
CA3029473
公开号:
CA3029473A1
申请日:
2017.06.28
申请国别(地区):
CA
年份:
2018
代理人:
摘要:
Disclosed herein are compositions and formulations containing a TAT?-CDKL5 fusion protein. Also disclosed are methods of producing a TAT?-CDKL5 fusion protein from vectors containing a TAT?-CDKL5 cDNA and methods of transducing cells with the vectors containing a TAT?-CDKL5 cDNA and the TAT?-CDKL5 fusion protein. Also disclosed are uses of TAT?-CDKL5 fusion proteins for treating CDKL5 deficiencies by systemic or intravenous administration of the fusion proteins.