LNP-si-SCR

LNP-si-SCR is a research-grade lipid nanoparticle (LNP) formulation containing a non-targeting, scrambled small interfering RNA (si-SCR). It serves as a negative control in preclinical studies to evaluate the efficacy and specificity of siRNA-based therapies, particularly those delivered via LNPs. The formulation described in the context utilizes a lipid composition similar to the FDA-approved drug Onpattro (patisiran), including D-Lin-MC3-DMA, DSPC, cholesterol, and PEG-DMG. In studies of pediatric acute myeloid leukemia (AML), LNP-si-SCR was employed to demonstrate that the silencing of the long non-coding RNA LINC01257 was specific to the targeted siRNA and not a result of the delivery vehicle or non-specific RNA effects. The nanoparticles typically have a diameter of approximately 65 nm and high siRNA encapsulation efficiency (>85%).

Other names

si-SCR LNPscrambled siRNA lipid nanoparticleLNP-siRNA control