siRNA PMP22-SQ NPs

siRNA PMP22-SQ NPs is a preclinical siRNA-based therapy developed for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). The therapy utilizes small interfering RNA (siRNA) designed to target and knock down the expression of the PMP22 gene, which is overexpressed in CMT1A patients due to a genetic duplication. To ensure effective delivery, the siRNA is conjugated to squalene nanoparticles (SQ NPs), which protect the genetic material from enzymatic degradation and enhance cellular uptake. Preclinical research in transgenic mouse models has shown that intravenous administration of this therapy can normalize PMP22 protein levels, restore motor function and electrophysiological parameters, and promote the regeneration of myelin and axons. The development of this program involved French academic institutions such as Inserm and Université Paris Saclay, with funding and partnership support from the Charcot-Marie-Tooth Research Foundation and MAASiRNA.

Other names

siRNA-squalene nanoparticlesPMP22 siRNA squalene conjugatePMP-22 siRNA squalene conjugatePMP 22 siRNA squalene conjugatesiRNA-SQ NPs