Quantifying mutant huntingtin protein (mHTT) – Two independent validations of a mHTT assay to support the clinical development of mHTT-targeting therapies in Huntington’s disease
The most advanced therapies under development for Huntington’s disease (HD) currently focus on lowering levels of mutant huntingtin protein (mHTT) in the brain. The optimisation and validation of a bioanalytical method for the reliable quantification of mHTT levels in the cerebrospinal fluid (CSF) of individuals with HD will facilitate the regulatory approval of a disease-modifying therapy for HD. This study reports the results of two independent validations of an assay designed for the relative quantification of mHTT in human CSF.
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