发明名称 METHOD OF REGULATING SMN GENE EXPRESSION
摘要 <p>The invention relates to methods and compositions for regulating the expression of the survival motor neuron gene. The invention also relates to nucleic acid constructs that can be used to regulate gene expression in cells, in vitro, ex vivo or in vivo. The invention is particularly concerned with the use of compounds capable of increasing the levels of an IRF or Stat factor within a cell, such as interferons or neurotrophic factors, for the preparation of a composition for the regulation of SMN and/or SMNc gene expression. The invention can be used to produce various proteins or peptides, as well as for the treatment of SMA.</p>
申请公布号 WO2000021553(A2) 申请公布日期 2000.04.20
申请号 EP1999007544 申请日期 1999.10.08
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