Table 1.
Designs, Sequences, and Target Messenger RNAs of the Small Interfering RNAs Used in This Study
| Compound | Strand | Sequence (5′–3′) | Target | Target accession number |
|---|---|---|---|---|
| AD-60519 | S | c•a•gaaaGaGuGuCuCaucuuaL | ALAS1 | NM_000688 |
| AS | u•A•AGaUgAgAcAcUcUuUcUg•g•u | |||
| siRNA2 | S | Proprietarya | AAT | NM_001127701 |
| AS | Proprietarya | |||
| AD-65644 | S | g•a•auguGaaAGucaucgacaaL | HAO1 | NM_017545 |
| AS | u•U•gucGaUGacuuUcAcauuc•u•g | |||
| AD-64543 | S | c•a•CuGuGaCUGuGgCcUcCaAL | TMPRSS6 | NM_153609 |
| AS | u•U•gGaGgCcAcagUcAcAgUg•c•u | |||
| AD-68435 | S | u•g•ugcaAuGAAaggcaaauauL | F9 | NM_000133 |
| AS | a•U•auuUgCCuuucAuUgcaca•c•u |
L
S and AS represent sense and antisense strands; uppercase and lowercase letters indicate 2′-deoxy-2′-fluoro (2′-F) and 2′-O-methyl (2′-OMe) ribosugar modifications, respectively.
a
Design similar to GalNAc-conjugated siRNAs described in this table; • indicates phosphorothioate linkage; L indicates the trivalent GalNAc ligand (structure above).
AAT, alpha-1 antitrypsin; ALAS1, aminolevulinic acid synthase; F9, coagulation factor IX; GalNAc, N-acetylgalactosamine; HAO1, hydroxy acid oxidase 1; siRNA, small interfering RNA; TMPRSS6, transmembrane serine protease 6.