Researchers from Qingdao University Report Recent Findings in siRNA-Based Therapy [Novel Diamine-scaffold Based n-acetylgalactosamine (Galnac)-sirna Conjugate: Synthesis and in Vivo Activities].

Researchers from Qingdao University in the People's Republic of China have developed a novel diamine-scaffold GalNAc conjugate for siRNA administration in gene therapy. The conjugate has shown comparable siRNA delivery effectiveness to a GalNAc NAG37 phase II clinical drug candidate and exhibits more powerful silencing activity when connected to the 3'-end of the sense strand with an additional PS-linkage. The incorporation of a diamine-scaffold into the GalNAc conjugate structure shows potential in the field of gene therapy. This research was supported by the Shandong Provincial Postdoctoral Science Foundation. [Extracted from the article]