Treatment typically begins with a starting dose of 0.25 mg once weekly, followed by a gradual dose escalation schedule
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Mechanistic studies using CRISPR/Cas9-engineered cell lines bearing the C65G mutation revealed a multilayered disruption of tRNA [Ser]Sec function: reduced tRNA expression, severe impairment of selenoprotein synthesis, and most strikingly a loss of selenium responsiveness not observed with other tRNA [Ser]Sec mutants [111]
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