Title:
GENE THERAPY FOR NEURONAL CEROID LIPOFUSCINOSES
Document Type and Number:
Japanese Patent JP2023099113
Kind Code:
A
Abstract:
To provide methods and compositions for treatment of Batten disease.SOLUTION: Such compositions include a recombinant adeno-associated virus (rAAV), the rAAV comprising an AAV capsid, and a vector genome packaged therein, the vector genome comprising (a) an AAV 5' inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3' ITR.SELECTED DRAWING: None
Inventors:
JAMES M WILSON
KATZ NATHAN
CHRISTIAN HINDERER
JULIETTE HORDEAUX
KATZ NATHAN
CHRISTIAN HINDERER
JULIETTE HORDEAUX
Application Number:
JP2023073941A
Publication Date:
July 11, 2023
Filing Date:
April 28, 2023
Export Citation:
Assignee:
UNIV PENNSYLVANIA
International Classes:
C12N7/01; A61K9/10; A61K9/51; A61K31/7088; A61K35/76; A61K47/69; A61K48/00; A61P25/00
Attorney, Agent or Firm:
Patent Attorney Corporation Odashima Patent Office
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