专利检索 ap:"Rando, Thomas A." 第 1 页

1.

发明公开
USE OF CHIMERIC MUTATIONAL VECTORS TO CHANGE ENDOGENOUS SEQUENCES IN SOLID TISSUES 审中-公开
标题翻译：嵌合突变载体变革的源序列中使用固定的组织

公开(公告)号：EP1178836A2

公开(公告)日：2002-02-13

申请号：EP00932605.9

申请日：2000-05-19

申请人： The Board of Trustees of The Leland S. Stanford Junior University , The University of Miami , Bartlett, Richard J. , Rando, Thomas A.

发明人： Bartlett, Richard J. , Rando, Thomas A.

IPC分类号： A61K48/00 , C12N15/90 , A61P21/00

CPC分类号： A61K48/00 , C12N15/907

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.