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HYBRID PROMOTERS AND THEIR USES IN THERAPY, NOTABLY FOR TREATING TYPE II COLLAGENOPATHIES

机译:杂交促进剂及其在治疗中的用途,特别是用于治疗II型胶原蛋白

摘要

The present invention relates to hybrid promoters with a specific design, comprising fragments of the hCOL2A1 promoter and fragments of the hEF1α promoter, which may be of interest in therapy, particularly in gene therapy. Notably, they may be introduced into a vector for expressing a nucleic acid sequence of interest. They may be particularly useful for treating skeletal dysplasia, such as type II collagenopathies; or articular diseases.
机译:本发明涉及具有特异性设计的杂化启动子,其包括HCOR2A1启动子的片段和HEF1α启动子的片段,其可能对治疗感兴趣,特别是在基因疗法中。 值得注意的是,可以将它们引入载体中以表达感兴趣的核酸序列。 它们对于治疗骨骼发育性特别有用,例如II型胶原蛋白病; 或关节疾病。

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