发明公开
EP1178836A2 USE OF CHIMERIC MUTATIONAL VECTORS TO CHANGE ENDOGENOUS SEQUENCES IN SOLID TISSUES
审中-公开
嵌合突变载体变革的源序列中使用固定的组织
- 专利标题: USE OF CHIMERIC MUTATIONAL VECTORS TO CHANGE ENDOGENOUS SEQUENCES IN SOLID TISSUES
- 专利标题(中): 嵌合突变载体变革的源序列中使用固定的组织
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申请号: EP00932605.9申请日: 2000-05-19
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公开(公告)号: EP1178836A2公开(公告)日: 2002-02-13
- 发明人: Bartlett, Richard J. , Rando, Thomas A.
- 申请人: The Board of Trustees of The Leland S. Stanford Junior University , The University of Miami , Bartlett, Richard J. , Rando, Thomas A.
- 申请人地址: 900 Welch Road, Suite 350 Palo Alto, CA 94304 US
- 专利权人: The Board of Trustees of The Leland S. Stanford Junior University,The University of Miami,Bartlett, Richard J.,Rando, Thomas A.
- 当前专利权人: The Board of Trustees of The Leland S. Stanford Junior University,The University of Miami,Bartlett, Richard J.,Rando, Thomas A.
- 当前专利权人地址: 900 Welch Road, Suite 350 Palo Alto, CA 94304 US
- 代理机构: Brasnett, Adrian Hugh
- 优先权: US135139P 19990521; US174388P 20000103
- 国际公布: WO0071165 20001130
- 主分类号: A61K48/00
- IPC分类号: A61K48/00 ; C12N15/90 ; A61P21/00
摘要:
This invention relates to the field of muscular dystrophy and methods for its treatment in humans. This invention also concerns art-recognized animal models of Duchenne muscular dystrophy in dogs (GRMD) and mice mdx Another aspect concerns chimeric mutational vector7 capable of inducing reversion of generic mutations (i.e., gene repair) causing genetic disease by direct injection into affected tissue. Thus, more generally, the invention envisions direct injection of chimeric mutational vectors into affected tissues to effect gene repair therein.
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