Knocking down disease: a progress report on siRNA therapeutics.

Forskningsoutput: TidskriftsbidragÖversiktsartikel

Abstract

Small interfering RNAs (siRNAs), which downregulate gene expression guided by sequence complementarity, can be used therapeutically to block the synthesis of disease-causing proteins. The main obstacle to siRNA drugs - their delivery into the target cell cytosol - has been overcome to allow suppression of liver gene expression. Here, we review the results of recent clinical trials of siRNA therapeutics, which show efficient and durable gene knockdown in the liver, with signs of promising clinical outcomes and little toxicity. We also discuss the barriers to more widespread applications that target tissues besides the liver and the most promising avenues to overcome them.

Detaljer

Författare
Enheter & grupper
Forskningsområden

Ämnesklassifikation (UKÄ) – OBLIGATORISK

  • Farmakologi och toxikologi
Originalspråkengelska
Sidor (från-till)543-552
TidskriftNature Reviews. Genetics
Volym16
Utgåva nummer9
StatusPublished - 2015
PublikationskategoriForskning
Peer review utfördJa