Oligonucleotide therapy for leber congenital amaurosis

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专利权人:

ProQR Therapeutics II B.V.

发明人:

Biasutto, Patricia Coromoto,Chan, Hee Lam

申请号：

AU2016223336

公开号：

AU2016223336A1

申请日:

2016.02.26

申请国别(地区):

AU

年份:

2017

代理人:

摘要：

Antisense oligonucleotides target the mutation in intron 26 of the CEP290 gene and reduce inclusion of the aberrant exon into the CEP290 mRNA. The oligonucleotides include no more than 3 consecutive guanosines, have no more than 60% guanosine nucleobases, include at most one CpG sequence, and/or do not have the potential to form a hairpin comprising 3 or more consecutive complementary base pairs.

来源网站：

中国工程科技知识中心

来源网址：

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