摘要 |
<p>The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.</p> |
申请人 |
ISIS PHARMACEUTICALS, INC.;BHANOT, SANJAY;GEARY, RICHARD, S.;MCKAY, ROBERT;MONIA, BRETT, P.;SETH, PUNIT, P.;SIWKOWSKI, ANDREW, M.;SWAYZE, ERIC, E.;WANCEWICZ, EDWARD |
发明人 |
BHANOT, SANJAY;GEARY, RICHARD, S.;MCKAY, ROBERT;MONIA, BRETT, P.;SETH, PUNIT, P.;SIWKOWSKI, ANDREW, M.;SWAYZE, ERIC, E.;WANCEWICZ, EDWARD |