Title:
ムコ多糖症ＩＶＡ型の治療
Document Type and Number:
Japanese Patent JP2021531044
Kind Code:
A
Abstract:
本発明で提供するのは、ムコ多糖症ＩＶＡ型（ＭＰＳ ＩＶＡ）を治療するための遺伝子療法の方法であって、組み換えアデノ随伴ウイルス（ｒＡＡＶ）を用いて、ＭＰＳ ＩＶＡと診断されたヒト対象の骨に、ヒトＮ−アセチルガラクトサミン−６−硫酸スルファターゼ（ｈＧＡＬＮＳ）を送達することを伴う方法である。また、本発明で提供するのは、その遺伝子療法の方法で使用できるｒＡＡＶと、そのようなｒＡＡＶの作製方法である。【選択図】図１
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Inventors:
Shunji Tomatsu
Kazuki Sawamoto
Subha Kalmtil Meletil
Olivier Danos
Kazuki Sawamoto
Subha Kalmtil Meletil
Olivier Danos
Application Number:
JP2021527020A
Publication Date:
November 18, 2021
Filing Date:
July 26, 2019
Export Citation:
Assignee:
Legends Bio Inc.
The Nemur Foundation
The Nemur Foundation
International Classes:
C12N15/62; A61K35/76; A61K38/14; A61K48/00; A61P1/00; A61P9/00; A61P11/00; A61P13/12; A61P19/00; A61P21/00; A61P43/00; C12N5/10; C12N7/01
Domestic Patent References:
| JP2016535587A | 2016-11-17 | |||
| JP2008501307A | 2008-01-24 | |||
| JP2018517414A | 2018-07-05 |
Foreign References:
| WO2019228950A1 | 2019-12-05 |
Other References:
TOMATSU, S. ET AL.: "Current and emerging treatments and surgical interventions for Morquio A syndrome: a review", RES REP ENDOCR DISORD, vol. 2012(2), JPN6023024825, December 2012 (2012-12-01), pages 65 - 77, ISSN: 0005087305
ALMECIGA-DIAZ, C. J. ET AL.: "Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modify", FEBS JOURNAL, vol. 277, JPN6023024824, 2010, pages 3608 - 3619, XP055501647, ISSN: 0005087306, DOI: 10.1111/j.1742-4658.2010.07769.x
ALMECIGA-DIAZ, C. J. ET AL.: "Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modify", FEBS JOURNAL, vol. 277, JPN6023024824, 2010, pages 3608 - 3619, XP055501647, ISSN: 0005087306, DOI: 10.1111/j.1742-4658.2010.07769.x
Attorney, Agent or Firm:
Toru Ishikawa
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