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ACIDE RIBONUCLÉIQUE DOUBLE BRIN PRÉSENTANT UNE EFFICACITÉ ACCRUE DANS UN ORGANISME
专利权人:
Alnylam Europe AG
发明人:
申请号:
EP19196267.9
公开号:
EP3604537A1
申请日:
2004.06.14
申请国别(地区):
EP
年份:
2020
代理人:
摘要:
The present invention elates to a double-stranded ribonucleic acid (dsRNA), wherein said dsRNA comprises an antisense RNA strand and a sense RNA strand, wherein said antisense RNA strand has a nucleotide sequence of less than 30 nucleotides in length and is substantially complementary to a target RNA, wherein (a) at least one end of said dsRNA comprises a 1 to 2 nucleotide overhang, (b) said dsRNA comprises at least one chemically modified nucleotide, wherein said at least one chemically modified nucleotide is a 2'-O-methyl nucleotide, (c) the unpaired nucleotide adjacent to the terminal nucleotide base pair comprises a purine base, (d) said dsRNA is conjugated to a ligand, wherein said ligand is selected from (i) carbohydrate clusters, (ii) polyethylene glycols, (iii) delivery peptides, (iv) crosslinking agents, and (v) porphyrin conjugates, (e) at least one nucleotide of the dsRNA comprises a phosphorothioate or phosphorodithioate group, and wherein substantially complementary means having at least 90% sequence identity.
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