Invention Grant
- Patent Title: Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly or abnormally expressed gene
- Patent Title (中): 通过修改异常或异常表达基因的表达来治疗个体遗传疾病的干预
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Application No.: US12297251Application Date: 2007-04-20
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Publication No.: US08304398B2Publication Date: 2012-11-06
- Inventor: Peter Abraham Christiaan 't Hoen , Petronella Johanna Elisabeth Sterrenburg , Johannes Theodorus den Dunnen , Garrit Jan Boudewijn van Ommen
- Applicant: Peter Abraham Christiaan 't Hoen , Petronella Johanna Elisabeth Sterrenburg , Johannes Theodorus den Dunnen , Garrit Jan Boudewijn van Ommen
- Applicant Address: NL Leiden
- Assignee: Academisch Ziekenhuis Leiden
- Current Assignee: Academisch Ziekenhuis Leiden
- Current Assignee Address: NL Leiden
- Agency: Edwards Wildman Palmer LLP
- Agent Kathleen Williams; Elizabeth Spar
- Priority: WOPCT/NL2006/000207 20060420
- International Application: PCT/NL2007/050175 WO 20070420
- International Announcement: WO2007/123402 WO 20071101
- Main IPC: A61K31/70
- IPC: A61K31/70 ; C07H21/04
Abstract:
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.
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