HTS in Search of RNA Splicing Modulators Using a HiBiT Assay

Cytoplasmic aggregation of mis-localized proteins is a common pathological hallmark in neurodegenerative disorders. Dysregulation of nuclear DNA binding proteins crucial for RNA stabilization, transcription, and splicing leads to aberrant gene expression, resulting in the accumulation of truncated variants. Recent findings directly associate these variants with severe neurological conditions. We developed a bioluminescent cell-based assay capable of mimicking this pathology through inducible protein depletion, utilizing HiBiT tagging of the gene of interest (GOI). Our fast, sensitive and simple assay enables high-throughput screening for RNA splicing modulators, offering a potential avenue to restore proper expression of the wild-type GOI isoform, and therefore of the wild-type protein. Join us for this webinar where Dr. Gene Ananiev from Promega will introduce the HiBiT epitope tag and its various advantages for high-throughput applications and Gianluca Conti from AXXAM, will illustrate the assay principle and will show the results obtained by an HTS campaign.