LSD1 inhibition ameliorates congenital dyserythropoietic anemia type II - PubMed
3 hours ago
- #CDAII therapy
- #LSD1 inhibition
- #SEC23A expression
- LSD1 inhibition rescues congenital dyserythropoietic anemia type II (CDAII) by increasing SEC23A expression.
- Small-molecule screen identified LSD1 inhibitor RN1 as top compound increasing SEC23A-eGFP abundance without impairing erythroid growth/differentiation.
- Genetic down-regulation or deletion of LSD1 increased SEC23A mRNA and expression, ameliorating erythroid defects in CDAII models.
- Mechanistically, LSD1 represses SEC23A transcription by occupying its promoter; deletion of this sequence rescued CDAII.
- Findings suggest LSD1 inhibition as a promising therapeutic strategy for CDAII.