Optimized CLN1 genes and expression cassettes and their use

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专利权人:

The University of North Carolina at Chapel Hill

发明人:

Gray, Steven

申请号：

AU2017284198

公开号：

AU2017284198A1

申请日:

2017.06.13

申请国别(地区):

AU

年份:

2018

代理人:

摘要：

This invention relates to polynucleotides comprising a nucleotide sequence encoding a PPT1 polypeptide or a fragment thereof, vectors (viral or non-viral vectors) comprising the same, and methods of using the same for delivery of the open reading frame to a cell or a subject and to treat infantile neuronal lipofuscinosis (infantile Batten disease). The polynucleotides comprise an optimized CLN1 open reading frame.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/