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Title:
METHODS AND COMPOSITIONS FOR TREATMENT OF LYSOSOMAL STORAGE DISEASES
Document Type and Number:
Japanese Patent JP2018038410
Kind Code:
A
Abstract:
PROBLEM TO BE SOLVED: To provide methods and compositions for treatment of lysosomal storage diseases.SOLUTION: The invention provides nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease. The invention describes a method for inserting a transgene sequence into a suitable target cell, where the transgene encodes a protein that treats the disease.SELECTED DRAWING: None

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Inventors:
EDWARD J REBAR
Application Number:
JP2017195993A
Publication Date:
March 15, 2018
Filing Date:
October 06, 2017
Export Citation:
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Assignee:
SANGAMO BIOSCIENCES INC
International Classes:
C12N5/10; A61K35/12; A61K35/18; A61K35/28; A61K35/34; A61K35/407; A61K35/545; A61K38/46; A61K38/47; A61K48/00; A61P3/00; A61P43/00
Foreign References:
WO2011100058A12011-08-18
WO2011097036A12011-08-11
Other References:
LANCET, 2008, 372(9645):1263-71, JPN6017001081, ISSN: 0003933103
ANGUELA, X. M. ET AL., BLOOD, 2012, VOL.120, NO.21, ABSTRACT NO.751, JPN6018047601, ISSN: 0003933104
SCIENCE, 2013 FEB, VOL.339, NO.6121, PP.819-822, JPN6018047602, ISSN: 0003933105
SCIENCE, 2013 FEB, VOL.339, NO, 6121, PP.823-826, JPN6018047603, ISSN: 0003933106
ELIFE, 2013 JAN, VOL.29, NO.2, E00471, PP.1-9, JPN6018047604, ISSN: 0003933107
Attorney, Agent or Firm:
Hidesaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto



 
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