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SINGLE-STRANDED RNA-EDITING OLIGONUCLEOTIDES
专利权人:
ProQR Therapeutics II B.V.
发明人:
Janne Juha Turunen,Petra Geziena De Bruijn,Bart Klein,Roxana Simona Redis,Lenka Van Sint Fiet
申请号:
US16309954
公开号:
US20190330622A1
申请日:
2017.06.22
申请国别(地区):
US
年份:
2019
代理人:
摘要:
The invention relates to antisense oligonucleotides that are capable of bringing about specific editing of a target nucleotide (adenosine) in a target RNA in a eukaryotic cell, wherein said oligonucleotide does not, in itself, form an intramolecular hairpin or stem-loop structure, and wherein said oligonucleotide comprises a cytidine (a non-complementary nucleotide) or a uridine in position opposite to the target adenosine to be edited in the target RNA region.
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