CLK1 exon 4 morpholino

CLK1 exon 4 morpholino is an experimental splice-switching antisense oligonucleotide (specifically a Vivo-Morpholino) designed to target the intron 3-exon 4 splice junction of the **CDC-like kinase 1 (CLK1)** pre-mRNA. By sterically blocking this junction, the drug induces the skipping of exon 4 during mRNA processing. Because exon 4 is essential for the production of the catalytically active kinase isoform of CLK1, its exclusion leads to a significant reduction in both CLK1 mRNA and protein levels. In pediatric brain tumor models, such as high-grade gliomas (HGG) and diffuse midline gliomas (DMG), depletion of CLK1 via this morpholino impairs cell growth and viability by disrupting oncogenic splicing programs and affecting pathways related to the cell cycle, DNA repair, and MAPK signaling. It is currently utilized as a research tool to validate CLK1 as a therapeutic dependency in aggressive central nervous system malignancies.

Other names

CLK1 exon 4 targeted morpholinoCLK-1 exon 4 targeted morpholinoCLK 1 exon 4 targeted morpholinoVivo-Morpholino targeting CLK1 exon 4CLK1 exon 4 skipping morpholinoCLK-1 exon 4 skipping morpholinoCLK 1 exon 4 skipping morpholino