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We validated in human trials, a platform for reducing the synthesis of genes expressed in the liver. The platform utilizes a GalNAc ligand attached to the 3’ end of the sense strand of an RNAi ...

This fundamental work presents two clinically relevant BMP4 mutations that contribute to vertebrate development. The compelling evidence, both from wet lab and AI generated predictions, supports that ...

(1) This approach is exemplified by givorisan and inclisiran, two FDA-approved small interfering RNAs (siRNAs) conjugated to a triantennary N-acetylgalactosamine (GalNAc) moiety, enabling hepatic ...

To address these disparities, it is imperative to adapt medical technologies and approaches to the diverse populations they aim to serve. By fostering inclusivity and considering the unique needs and ...

All study samples were kept frozen at −80 °C until analysis. To demonstrate our workflow, we employed four FDA-approved GalNAc-siRNAs: Givosiran, Lumasiran, Inclisiran, and Vutrisiran, in addition to ...