- 专利标题： COMPOSITIONS FOR TREATING FRIEDREICH'S ATAXIA
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申请号： PCT/US2020/066167申请日： 2020-12-18
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公开(公告)号： WO2021127533A1公开(公告)日： 2021-06-24
- 发明人: WILSON, James, M. , HINDERER, Christian , MILLER, Nimrod
- 申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
- 申请人地址： 3600 Civic Center Blvd
- 专利权人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
- 当前专利权人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
- 当前专利权人地址： 3600 Civic Center Blvd
- 代理机构： KODROFF, Cathy, A. et al.
- 优先权： US62/950,834 2019-12-19
- 主分类号： C12N15/864
- IPC分类号： C12N15/864 ; A61K35/76 ; C12P19/34 ; C12N15/79
摘要：
A recombinant adeno-associated virus (rAAV) comprising an AAV capsid and a vector genome comprising a frataxin gene is provided. Also provided is a composition containing an effective amount of rAAV to ameliorate symptoms of Freidreich's ataxia, including, e.g., reduction in progression towards neurocognitive decline and/or cardiomyopathy.
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