Monica N. Montoute

TL;DR: These findings demonstrate that the NOD2 signaling pathway is amenable to modulation by small molecules that do not target RIP2 kinase activity and should prove useful tools to investigate the importance of N OD2 in various inflammatory processes and may have potential clinical utility.

TL;DR: It is concluded that in these preclinical models, selective, reversible inhibition of DNMT1 is sufficient for the induction of HbF, and is well-tolerated.

TL;DR: Results indicate that the NOD1 signaling pathway, similarly to activation of NOD2, is amenable to modulation by small molecules that do not target RIP2 kinase, and should prove useful tools to investigate the importance of Nod1 activation in various inflammatory processes.

TL;DR: To identify compounds that may lead to the development of better and safer medicines, a method using primary human bone marrow day 7 erythroid progenitor cells (EPCs) to screen for compounds that induce γ-globin production is established.

TL;DR: A phenotypic screen in human erythroid progenitor cells resulted in the identification of the benzoxaborole-containing hit compound 1, which had approximately 100-fold improved cellular potency compared to 1, high levels of oral exposure in rats, and excellent solubility.