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ZhuXYinLTheisenMZhuoJSiddiquiSLevyBet alSystemic mRNA therapy for the treatment of fabry disease: preclinical studies in wild-type mice, fabry mouse model, and wild-type non-human Primates

CUT & Tag CUT & Tag was performed using the Hyperactive Universal CUT & Tag Assay Kit for Illumina Pro (Vazyme, TD904), according to the manufacturers instructions

Activated S6K1 facilitates the initiation of mRNA translation by phosphorylating substrates such as eIF4B and promoting the degradation of PDCD4, an inhibitor of eIF4B (134)

A combination of both is most effective

It is a devastating disease with a lack of effective treatments and although the cause of the neuronal death in ALS is not known, it is suspected that oxidative damage plays a key role