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While ERT can normalize biochemistry quickly, the need for chronic administration and the typical costs and immunogenicity risks of biologics remain practical challenges for widespread adoption (58)

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However, the efficiency of gene silencing by naked siRNA is very low, because the naked siRNA molecules are rapidly degraded by nucleases in the bloodstream and experienced rapid renal clearance in the body 19,20

Of note, the majority of these studies refer to the involvement of miRNAs that target genes tightly related with the GH/IGF1 axis

Dornier E., Rabas N., Mitchell L., et al., Glutaminolysis Drives Membrane Trafficking to Promote Invasiveness of Breast Cancer Cells, Nature Communications 8, no