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Application of saRNA and mRNA for Gaucher disease treatment as protein replacement therapy

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(Left) When self-amplifying RNA (saRNA) is injected and delivered into cells, the translation of replicase takes place. The replicase uses the saRNA as a template and makes a negative saRNA strand, which in turn serves as a template for self-amplification by the replicase. Replicase recognizes sub-genomic promoter in the negative strand, so sgRNA is synthesized. Consequently, high levels of glucocerebrosidase are produced. (Right) When the mRNA is injected and delivered into the cells, it is translated into glucocerebrosidase protein. Ultimately, the glucocerebrosidase produced breaks down the GlcCer into glucose and ceramide leading to the normal condition.

Abbreviations: saRNA, self-amplifying RNA; mRNA, messenger RNA; sgPr, sub-genomic promoter; GD, Gaucher disease; GCase, glucocerebrosidase; GlcCer, glucosylceramide.

(Source: Feng S, Rcheulishvili N, Jiang X, et al. A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach. Int J Biol Sci 2024;20[6]:2111-29. Creative Commons Attribution 4.0 International [CC BY 4.0] license, creativecommons.org/licenses/by/4.0.)