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OLIGONUCLEOTIDE THERAPY FOR LEBER CONGENITAL AMAUROSIS
专利权人:
ProQR Therapeutics II B.V.
发明人:
Patricia Coromoto Biasutto,Hee Lam Chan
申请号:
US16576853
公开号:
US20200063134A1
申请日:
2019.09.20
申请国别(地区):
US
年份:
2020
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.
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