912641-74-8

Upstream product

• 51224-10-3 1-iodo-6-(trifluoroacetamido)hexane 
• 40615-36-9 4,4'-dimethoxytrityl chloride 
• 42822-78-6 2,3’-O-dibutylstannyleneuridine 
• 383-63-1 ethyl trifluoroacetate, 
• 165381-00-0 5'-O-(dimethoxytrityl)-2'-O-(hexylamino)uridine 
• 51224-09-0 6-(2,2,2-trifluoroacetamido)hexyl 4-methylbenzenesulfonate 
• 40248-34-8 6-trifluoroacetamido-1-hexanol 
• 4048-33-3 6-amino-1-hexanol 

Relevant academic research and scientific papers

Zwitterionic oligonucleotides: A study on binding properties of 2′-O-aminohexyl modifications

2′-O-Aminohexyl side chains provide excellent conditions for zwitterionic interstrand and intrastrand interactions of oligonucleotides. 2′-O-Aminoalkylated phosphoramidites of adenosine and undine were synthesized and incorporated in increasing number int

EXTRAHEPATIC DELIVERY

One aspect of the present invention relates to a compound comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic monomers, containing one or more lipophilic moieties, conjugated to one or more positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic monomer-conjugated compound.

TRANSTHYRETIN (TTR) IRNA COMPOSITION AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED OCULAR DISEASES

The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR- associated ocular diseases.