Johannes Theodorus den Dunnen;Petronella Johanna Elisabeth Sterrenburg;Garrit Jan Boudewijn van Ommen;Peter Abraham Christiaan 't Hoen;Peter Abraham Christiaan t Hoen
发明人:
Peter Abraham Christiaan 't Hoen,Petronella Johanna Elisabeth Sterrenburg,Johannes Theodorus den Dunnen,Garrit Jan Boudewijn van Ommen,Peter Abraham Christiaan t Hoen
申请号：
US12297251
公开号：
US08304398B2
申请日:
2007.04.20
申请国别(地区):
US
年份:
2012
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.
