发明名称 |
NUCLEIC ACID MOLECULE, EXPRESSION CASSETTE, EXPRESSION VECTOR, EUKARYOTIC HOST CELL, INDUCTION METHOD OF RNA INTERFERENCDE IN EUKARYOTIC HOST AND USE OF THE NUCLEIC ACID MOLECULE IN THERAPY OF DISEASES INDUCED BY EXPANSION OF TRINUCLEOTIDE GAG REPEATS |
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. |
申请公布号 |
US2016376586(A1) |
申请公布日期 |
2016.12.29 |
申请号 |
US201414916039 |
申请日期 |
2014.09.02 |
申请人 |
INSTYTUT CHEMII BIOORGANICZNEJ PAN |
发明人 |
KRZYZOSIAK Wlodzimierz;OLEJNICZAK Marta;GALKA-MARCINIAK Paulina;FISZER Agnieszka |
分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
Poznan PL |