Alfica Sehgal

TL;DR: In this paper, a tetrahedral DNA strand self-assembles into tetrahedron nanoparticles that can deliver small interfering RNA molecules to cells and suppress genes in tumours.

TL;DR: A therapeutic approach mediated by RNA interference (RNAi) could reduce the production of transthyretin in peripheral nerves and the heart as mentioned in this paper, which is caused by the deposition of hepatocyte-derived tranthymretin amyloid in peripheral nerve and heart.

TL;DR: Treatment with ALN-AT3 promoted hemostasis in mouse models of hemophilia and led to improved thrombin generation in an NHP model of hemophile A with anti-factor VIII inhibitors.

TL;DR: Liver exposure data indicate that the improvement in potency is predominantly due to increased metabolic stability of the siRNA conjugates, and this work employed an iterative screening approach across multiple siRNAs to arrive at advanced designs with low 2′-deoxy-2′-fluoro content that yield significantly improved potency and duration in preclinical species, including non-human primate.

TL;DR: Compared two siRNAs of the same sequence but with different modification pattern resulting in different degrees of protection against nuclease activity enhanced stability translated into substantially improved liver exposure, gene silencing efficacy and duration of effect in mice.