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Methods and Compositions for Treatment of Muscular Dystrophy
专利权人:
TAWNY NEAL;JONATHAN M. GEISINGER;Alfonso P. FARRUGGIO;CHRISTOPHER L. CHAVEZ;MARISA KAROW;CHRISTOPHER BJORNSON;HASSAN CHAIB;MICHELE P. CALOS;CHUNLI ZHAO
发明人:
MICHELE P. CALOS,MARISA KAROW,CHRISTOPHER L. CHAVEZ,Alfonso P. FARRUGGIO,CHUNLI ZHAO,HASSAN CHAIB,CHRISTOPHER BJORNSON,TAWNY NEAL,JONATHAN M. GEISINGER
申请号:
US13310635
公开号:
US20120141441A1
申请日:
2011.12.02
申请国别(地区):
US
年份:
2012
代理人:
摘要:
The present disclosure provides methods for introducing a gene encoding a muscle membrane protein into a cell isolated from a subject to generate a genetically modified cell. The genetically modified cell may be introduced back, e.g., engrafted into the subject. The isolated cell may be additionally modified by introducing into the isolated cell a gene encoding one or more reprogramming transcription factors that induce the cell to form an induced pluripotent stem cell. The genetically modified cell may be differentiated in vitro to form muscle cell precursors before engrafting into the subject. Also provided are compositions comprising autologous cells isolated from a subject which cells comprise a muscle membrane protein gene integrated into a genome attachment site in the genome of the cell. The autologous cell may be an induced pluripotent cell or a mesenchymal stem cell, such as an adipose-derived mesenchymal stem cell (AD-MSC).
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