Genomic regulatory elements, such as enhancers and promoters, coordinate the binding and activity of TFs to drive transcription in human cells. Most enhancers contain binding sites for multiple TFs ...

A major hallmark of Parkinson's disease is loss of dopaminergic neurons in the substantia nigra pars compacta (SNpc). The pathophysiological mechanisms causing this relatively selective ...

The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector ...