A small-molecule screen reveals novel modulators of MeCP2 and X-chromosome inactivation maintenance.

Hyeong-Min LeeM Bram KuijerNerea Ruiz BlanesEllen P ClarkMegumi AitaLorena Galiano ArjonaAgnieszka KokotNoah SciakyJeremy M SimonSanchita BhatnagarBenjamin D PhilpotAndrea Cerase

Published in: Journal of neurodevelopmental disorders (2020)

Our results suggest that inhibition of the JAK/STAT pathway is a new potential pathway to reinstate MeCP2 gene expression as an efficient RTT treatment.

Keyphrases

small molecule
gene expression
protein protein
dna methylation
high throughput
risk assessment
combination therapy