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A therapy targeting directly the mRNA

We designed tailored siRNA sequences targeting specific mRNA. This innovation allows an effective modulation of gene expression at exceptionally low doses.

A revolutionary platform that delivers siRNA directly to Schwann cells  

We have pioneered a platform that leverages the conjugation of siRNA with a natural, biocompatible, and biodegradable lipid known as squalene. Squalene possesses the unique ability to self-assemble into nanoparticles in aqueous environments. These siRNA-squalene nanoparticles exhibit the ideal characteristics for crossing cell membranes and directly targeting damaged tissues following intravenous administration.

Schéma SHARK Squalene

A targeted therapy for CMT1A applicable to the other demyelinating diseases

MAAsiRNA is developing a new targeted therapy for CMT1A based on squalene siRNA nanoparticles.
Our therapeutic approach is specific and effective at low doses. In three transgenic CMT1A mouse models, treatment with siRNA-squalene nanoparticles normalized overexpression of the targeted mRNA, restored the nerve conduction velocity and rehabilitated the motor activity and strength of the mice (Boutary et al., 2021). No toxic effects were observed.

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