Alma Mater Studiorum - Università di Bologna;Amicus Therapeutics, Inc.
发明人:
CIANI, Elisabetta,LACCONE, Franco,CLARK, Sean
申请号:
EP17821132
公开号:
EP3475418A4
申请日:
2017.06.28
申请国别(地区):
EP
年份:
2020
代理人:
摘要:
Disclosed herein are compositions and formulations containing a TATĸ-CDKL5 fusion protein. Also disclosed are methods of producing a TATĸ-CDKL5 fusion protein from vectors containing a TATĸ-CDKL5 cDNA and methods of transducing cells with the vectors containing a TATĸ-CDKL5 cDNA and the TATĸ-CDKL5 fusion protein. Also disclosed are uses of TATĸ-CDKL5 fusion proteins for treating CDKL5 deficiencies by systemic or intravenous administration of the fusion proteins.