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

机译:混合启动子及其在治疗中的用途,尤其是用于治疗II型Collagenopathies

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:本发明涉及具有特定设计的杂合启动子,其包括hCOL2A1启动子的片段和hEF1α启动子的片段,这在治疗中,特别是在基因治疗中可能是有意义的。特别地,可以将它们引入表达目的核酸序列的载体中。它们对于治疗骨骼发育异常,例如II型胶原病尤其有用。或关节疾病。

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