INTERVENTION THERAPEUTIQUE DANS UNE MALADIE GENETIQUE CHEZ UN INDIVIDU PAR MODIFICATION DE L'EXPRESSION D'UN GENE EXPRIME DE MANIERE ABERRANTE OU ANORMALE
'T HOEN, PETER ABRAHAM CHRISTIAAN,STERRENBURG, PETRONELLA JOHANNA ELISABETH,VAN OMMEN, GARRIT-JAN BOUDEWIJN,DEN DUNNEN, JOHANNES THEODORUS
申请号：
CA2650229
公开号：
CA2650229C
申请日:
2007.04.20
申请国别(地区):
CA
年份:
2018
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.
