Huntington’s Disease Studies

We offer a comprehensive portfolio of validated in vitro and ex vivo cell based assays. These in vitro assays were developed specifically to screen RNAi and small molecules to novel mHTT regulators, identify small molecules for target degradation, optimize selective CNS penetrant ATM inhibitors, and other strategic therapeutic MOA’s.

This figure displays high content imaging of iPSC stem cells into striatal and cortical neurons

Cellular Models of Huntington’s Disease

This poster displays the differentiation of iPSC into striatal and cortical neurons.

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Stem Cell-Derived Striatal Neurons for In Vivo Huntington’s Disease Studies

Mariangela Iovino, PhD, Group Leader of Integrated Biology at Charles River presents at Neuroscience Day 2020 and takes a deep dive in to current Huntington’s disease research with stem cell-derived striatal neurons. View additional Neuroscience Day 2020 presentations here.


Huntingtin Detection Assay Using MSD Assay Platform

These in vitro assays were developed using the Meso Scale Discovery (MSD) technology. This technology allows for the quantification of HTT in many forms; full length, fragmented and aggregated HTT, from various species and against a variety of capture/detection antibodies.

Graph showing the increase in aggregated detection levels in the brain of Q175HET mice

Huntingtin Detection Assays

Aggregated HTT in the brain of Q175HET mice.

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HD Biomarker Detection

Our in vitro assays for the ultrasensitive detection of HD Biomarkers can be obtained using the Quanterix Simoa HD-1 platform. Assays have been validated to detect HTT levels using capture antibodies and Human Neurofiliment-Light(NfL) as an HD marker.

Results of primate CSF samples

Ultrasensitive Detection of Huntington’s Disease Biomarkers

MAB2166/2B7 HTT Assay

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