CSE1L shRNA

CSE1L shRNA is a short hairpin RNA-based therapeutic candidate and research tool designed to silence the expression of the CSE1L (Chromosome Segregation 1 Like) gene. CSE1L, also known as CAS (Cellular Apoptosis Susceptibility protein), is a nuclear exportin that plays a critical role in recycling importin-alpha and has been identified as a key regulator of ribosome biogenesis, specifically facilitating the export of the large ribosomal subunit (pre-60S) from the nucleus to the cytoplasm. Research indicates that CSE1L is significantly upregulated in childhood and young adult acute myeloid leukemia (AML) compared to older patients, representing a selective dependency in these populations. Treatment with CSE1L shRNA leads to the nuclear accumulation of ribosomal proteins, impaired rRNA maturation, and reduced leukemic cell viability and clonogenicity, while sparing normal hematopoietic cells, suggesting its potential as a targeted therapy for pediatric and young adult AML.

Other names

shRNA targeting CSE1LCAS shRNA