Christopher R. Vakoc

TL;DR: The data show that small-molecule inhibition of BRD4 has strong anti-leukemic effects in a broad range of AML subtypes, and it is shown that JQ1 synergizes with Ara-C in inducing growth inhibition in HL60 cells and KG-1 cells, and the results support the notion that J Q19s ability to suppress transcriptional modules may translate into a therapeutic entry point for eradicating LSC.

TL;DR: The preclinical studies that have linked BET proteins to the pathogenesis of hematopoietic malignancy and the confluence of evidence suggesting that suppression of MYC expression underlies, at least in part, these therapeutic effects are reviewed.

TL;DR: Two studies showed that some cancers are driven by genetic changes in the NSD3 protein that alter its enzymatic activity as discussed by the authors, which hints at a route to pharmacological reversal.

TL;DR: In humans, loss of the fragile X mental retardation protein, FMRP, leads to the most common inherited form of intellectual disability, the fragileX syndrome.

TL;DR: This study provides evidence that DNA hypermethylation and transcriptional silencing of ALDH2 occur in a recurrent manner in human AML patient samples, which is sufficient to confer FA pathway dependency in this disease.