Charles River Presents: The Innovator Series
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Platforms and Technologies
Utilizing vast compound libraries, chemists use high-throughput screening to rapidly identify promising compounds for neuroscience target validation and identification.
ASO screens can be designed to evaluate a variety of proven mechanism of action approaches for antisense oligonucleotide therapy. To date, we’ve developed ASO screening assays primarily for rare neurodegenerative disorders.
Modern medicinal chemistry is highly multidisciplinary, driving CNS drug discovery innovation from novel synthesis and delivery to screening. Small molecule and large molecule CNS targets range from GPCRs to kinase inhibitors and have proven useful for studying diseases of neurodegeneration such as Parkinson’s disease and Huntington’s disease.
In CNS research, we use high content imaging to characterize stem cell-derived neuronal lineages and a host of validated assays like apoptosis, autophagy, protein aggregation, cell or mitochondrial motility and migration, epigenetic modifications, maker expression, protein acetylation and phosphorylation, receptor internalization and degradation, sub-cellular localization and translocation of transcription factors.
Generating induced pluripotent stem cells (IPSCs) for neurological disease-relevant assays, coupled with specific differentiation protocols for production of neurons and/or astrocytes, improves translation to in vivo models.
CRISPR Cas9 gene editing has transformed drug discovery and development. It has been used successfully for editing the Huntington CAG repeat in human iPSCs and for high-content screening of disease-relevant in vitro assays for ALS.
Neurological disease research and drug discovery uses proteomics to search for disease or pharmacological signatures. This allows us to understand biological mechanisms and identify specific proteins and their modifications, e.g., ubiquitination or phosphorylation in Parkinson’s disease and Huntington’s disease models.
Our ion channel assays guide your early screening investigations and selectivity profiling with 120 targets organized in functionally-validated disease areas. CNS-focused Channel Panels™ include pain, psychiatric disorders, neurodegeneration, and seizure disorders.