Misfolding and aggregation of serum wild-type (wt) and mutant transthyretin protein (TTR) is the cause of Transthyretin Amyloidosis (ATTR), a severe and fatal disease characterized by abnormal deposits of TTR protein in the form of amyloid in the body’s organs and tissues. Currently new drugs are being developed which decrease the amount of TTR production as a mechanism to potentially slow or stop disease progression. Therefore, quantitation of TTR levels in serum is crucial in monitoring the pharmacodynamic efficacy of therapeutic measures. Conventional immunoturbometric or ELISA based assays of TTR do not differentiate between wt and mutant TTR. Presented is the application of MSIA for the simple and effective simultaneous quantification of wt and mutant TTR in human serum for monitoring the efficacy of a new therapeutic antisense drug.
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