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Title:
TATĸ-CDKL5 FUSION PROTEINS, COMPOSITIONS, FORMULATIONS, AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2018/005617
Kind Code:
A3
Abstract:
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.

Inventors:
CIANI ELISABETTA (IT)
LACCONE FRANCO (AT)
CLARK SEAN (US)
Application Number:
PCT/US2017/039692
Publication Date:
February 08, 2018
Filing Date:
June 28, 2017
Export Citation:
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Assignee:
ALMA MATER STUDIORUM UNIVERSITÁ DI BOLOGNA (IT)
AMICUS THERAPEUTICS INC (US)
International Classes:
C12N9/12; A61P25/00; A61P25/28; C12N9/96
Domestic Patent References:
WO2017153834A12017-09-14
Foreign References:
US20150247134A12015-09-03
US20160194617A12016-07-07
Other References:
TROPEA, D ET AL.: "Partial reversal of Rett Syndrome-like symptoms in MeCP2 mutant mice", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE U.S.A., vol. 106, no. 6, 10 February 2009 (2009-02-10), pages 2029 - 2034, XP055122225
Attorney, Agent or Firm:
ALEGRIA, Rory P. (US)
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