Title:
ライソゾーム病の遺伝子治療
Document Type and Number:
Japanese Patent JP7254815
Kind Code:
B2
Abstract:
The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof, Lysosomal Membrane Protein 2 (LIMP2), Prosaposin, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.
Inventors:
Averiobic, Artha
Heckman, Laura
Line, Elbe
Heckman, Laura
Line, Elbe
Application Number:
JP2020540682A
Publication Date:
April 10, 2023
Filing Date:
October 03, 2018
Export Citation:
Assignee:
PREVAIL THERAPEUTICS, INC.
International Classes:
C12N15/56; A61K31/7088; A61K35/76; A61K45/00; A61K48/00; A61P25/16; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N15/113; C12N15/12; C12N15/63; C12N15/864; C12N15/866
Domestic Patent References:
| JP2016503405A | ||||
| JP2004514407A | ||||
| JP2004516016A |
Foreign References:
| WO2016081927A2 | ||||
| US20030133924 |
Other References:
Protein Expression and Purification,2002年,vol. 26,p. 96-105
Journal of Virology,1997年,Vol.71, No.4,pp.3077-3082
Journal of Virology,1997年,Vol.71, No.4,pp.3077-3082
Attorney, Agent or Firm:
Kiyokazu