首页> 外国专利> NUCLEIC ACID MOLECULE, EXPRESSION CASSETTE, EXPRESSION VECTOR, EUKARYOTIC HOST CELL, INDUCTION METHOD OF RNA INTERFERENCE IN EUKARYOTIC HOST AND USE OF NUCLEIC ACID MOLECULE IN THERAPY OF DISEASES INDUCED BY EXPANSION OF TRINUCLEOTIDE CAG REPEATS

NUCLEIC ACID MOLECULE, EXPRESSION CASSETTE, EXPRESSION VECTOR, EUKARYOTIC HOST CELL, INDUCTION METHOD OF RNA INTERFERENCE IN EUKARYOTIC HOST AND USE OF NUCLEIC ACID MOLECULE IN THERAPY OF DISEASES INDUCED BY EXPANSION OF TRINUCLEOTIDE CAG REPEATS

机译:核酸分子,表达盒,表达载体,真核宿主细胞,RNA干扰在真核宿主中的诱导方法以及核酸分子在三聚体肽重复形成引起的疾病治疗中的应用

摘要

Subjects of the invention are: nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG-type repeats. Solution relates to the new concept of treating hereditary human neurological diseases caused by expansion of CAG-type trinucleotide repeats using RNA interference technology.
机译:本发明的主题是:核酸分子,表达盒,表达载体,真核宿主细胞,在真核宿主中RNA干扰的诱导方法以及该核酸分子在治疗由三核苷酸CAG型重复序列扩增引起的疾病中的用途。解决方案涉及使用RNA干扰技术治疗由CAG型三核苷酸重复序列扩展引起的遗传性人类神经系统疾病的新概念。

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