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:

Steven Gray

申请号：

US16305337

公开号：

US20200316222A1

申请日:

2017.06.13

申请国别(地区):

US

年份:

2020

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.

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