INTERVENTION THÉRAPEUTIQUE DANS UNE MALADIE GÉNÉTIQUE CHEZ UN INDIVIDU PAR MODIFICATION DE L'EXPRESSION D'UN GÈNE EXPRIMÉ DE MANIÈRE ABERRANTE OU ANORMALE

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专利权人:

Academisch Ziekenhuis Leiden

发明人:

申请号：

EP07747399.9

公开号：

EP2012813B1

申请日:

2007.04.20

申请国别(地区):

EP

年份:

2017

代理人:

摘要：

The present invention provides means and methods for alleviating genetic disease. A genetic defect that has a phenotype in differentiated cells can lead to defects in precursor cells thereof. These so-called secondary defects contribute to the overall disease of the individual. In the present invention, genetic intervention with the aim to alleviate symptoms of genetic disease is directed toward the primary genetic defect in the differentiated cell and the secondary defect in the precursor cell.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/