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Oligonucleotide therapy for leber congenital amaurosis
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/

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