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MOLÉCULES DE TRANS-ÉPISSAGE DE PRÉ-ARNM (RTM) PERFECTIONNÉES ET LEURS UTILISATIONS
专利权人:
BAUER; JOHANN
发明人:
申请号:
EP09802455.7
公开号:
EP2320952B1
申请日:
2009.07.30
申请国别(地区):
EP
年份:
2016
代理人:
摘要:
The present invention relates to specific and markedly improved pre-mRNA trans-splicing molecule (RTM) molecules which are designed to correct specific genes expressed within cells to be targeted, and which are associated with epidermolysis bullosa, cystic fibrosis, pachyonychia congenital, and psoriasis or neurodermitis, as well as cancers of the skin. In particular, the RTMs of the present invention are genetically engineered to interact with a specific target pre-mRNA expressed in cells to be targeted so as to result in correction of genetic defects or reprogramming of gene expression responsible for a variety of different skin disorders.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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