Protein production is an early biomarker for RNA-targeted therapies.
Wade K SelfKathleen M SchochJacob AlexNicolas BarthélemyJames G BollingerChihiro SatoTracy ColeHolly B KordasiewiczEric SwayzeRandall J BatemanTimothy M MillerPublished in: Annals of clinical and translational neurology (2018)
Measures of newly generated protein show earlier pharmacodynamics changes for RNA-lowering therapeutics compared with total protein concentration. Early in ASO treatment, decreases in newly generated protein are driven by changes in newly synthesized protein. Measuring new protein production in CSF may be a promising early pharmacodynamics marker for RNA-targeted therapeutics.