Title:
フリードライヒ運動失調症の治療用のベクター
Document Type and Number:
Japanese Patent JP7466855
Kind Code:
B2
Abstract:
The present invention provides gene therapies for the treatment of Friedreich's ataxia. Specifically, the present invention provides a nucleic acid, cloning vector and transfer vector for the production of an adeno-associated virus (AAV) vector. The nucleic acid comprises (i) a nucleic acid sequence encoding frataxin, (ii) a phospho-glycerate-kinase (PGK) promoter, and (iii) a woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The present invention also provides a pharmaceutical composition which comprises the AAV vector or nucleic acid. Also, the AAV vector, nucleic acid or pharmaceutical composition can be used as a medicament, specifically as a medicament for the treatment of Friedreich's ataxia.
Inventors:
Matilla Duenas Antoni
Sanchez Diaz Iberis
Balague Cabaces Eudaldo
Sanchez Diaz Iberis
Balague Cabaces Eudaldo
Application Number:
JP2022127545A
Publication Date:
April 15, 2024
Filing Date:
August 10, 2022
Export Citation:
Assignee:
Fundacio Institute Dinvestigacio Encience de la Salle Germans Trias Eye Puyol
Gentec, S.A.
Gentec, S.A.
International Classes:
C12N15/864; A61K35/76; A61K48/00; A61P9/00; A61P21/00; A61P25/00
Foreign References:
| WO2009034127A1 | ||||
| WO2016150964A1 | ||||
| WO2016115503A1 |
Attorney, Agent or Firm:
Saegusa International Patent Office
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