Nucleic acids, peptides and methods

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专利权人:

Fundació Centre de Regulació Genòmica

发明人:

Mark Isalan

申请号：

GB201519584

公开号：

GB2544270A

申请日:

2015.11.05

申请国别(地区):

GB

年份:

2017

代理人:

摘要：

Zinc finger polypeptides comprising from 8 to 23 zinc finger domains. The polypeptides may also comprise the human KRAB repressor domain from Kox-1 or the mouse KRAB repressor domain from ZF87. Also claimed is an adeno-associated virus (AAV) vector comprising a nucleic acid expression construct capable of expressing the zinc finger polypeptides. The polypeptides exhibit reduced immunotoxity in vivo and may have utility in sustained (mid- or long-term) expression in vivo and may be use in treating polyglutamine diseases such as Huntingtons disease.

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