Over the past two decades, the idea of targeting transcription factors to combat malignancies has turned into a clinical reality. Targeting oncogenes and their interactive partners is an effective approach to developing novel targeted therapies for cancer and other chronic diseases. The MYC family of proteins, which are transcription factors, play a pivotal role in many cellular processes. However, dysregulation of MYC, such as amplification of MYCN, is associated with tumorigenesis, especially for neuroblastoma. MDM2, on the other hand, is one of the most frequently studied oncogenes and is an excellent target for cancer therapy, based on its p53-dependent and p53-independent oncogenic activities in various cancers. 

A new study, led by USC Mark and Mary Stevens Neuroimaging and Informatics Institute (Stevens INI), will explore structural alterations in the brains of people with bipolar disorder (BD), a chronic mental illness with one of the highest rates of attempted suicide — and for which no biological tools currently exist to guide diagnosis or treatment. The goal is to transform researchers’ understanding of the disease in the hopes of developing more effective treatments. Christopher R.K. Ching, PhD, assistant professor of research neurology at the Stevens INI, part of the Keck School of Medicine of USC, will lead a global network of psychiatric researchers in collaboration with Matthew Kempton, PhD, of King’s College London. The NIH-funded project supports efforts by the Enhancing Neuro Imaging Genetics through Meta-Analysis (ENIGMA) Consortium’s Bipolar Disorder Working Group (ENIGMA-BD), which Ching chairs. The team will use a large-scale analysis approach called voxel-based morphometry (VBM), which allows scientists to map subtle structural alterations across the entire brain. Unlike other neuroimaging methods that tend to average features across larger predefined brain regions, this technique enables precise, fine-resolution mapping of the emotion and reward processing centers affected in BD and other regions like the cerebellum often overlooked in prior studies. 

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy and bone marrow transplantation – to alleviate symptoms of multiple sulfatase deficiency (MSD), an ultra-rare genetic disease with no cure. The studies, carried out in mice, not only offer new hope to children with MSD and their families but can also help researchers better understand common diseases with related genetic mutations.