About our lab
The Mandel lab applies a diverse set of approaches to questions related to the mechanisms of gene regulation in the healthy and diseased brain. At one end of a spectrum, we use animal models to study the developing and adult mouse brain, as well as to model the severe neurological disorder, Rett Syndrome, to better understand its molecular basis and how we might reverse symptoms. We also use human cell lines and brain tissue to study aging and its relationship to cognitive decline. At the extreme other end of the spectrum, we use high resolution imaging and single cell chromatin and transcriptomic analyses to unravel fundamental principles of gene regulation that extend across all organisms. This wide berth—from molecule to organism—allows us to capture the excitement of biology at more than one level and keeps our research at the forefront of the field.
Check out the lab's recent paper in Cell Reports
In vivo repair of a protein underlying a neurological disorder by Sinnamon et al., Cell Reports, 2020, is the first report showing success of an RNA editing approach, using Adeno Associated Virus delivery to the hippocampus, to restore protein function in neurons affected in a human disease. The work focuses on a patient loss-of-function mutation in the transcription factor, MeCP2, which underlies the neurological disorder, Rett syndrome. Repairing the mutation leads to recovery of chromatin binding of MeCP2 across three different neuronal populations in the hippocampus in the affected mice. Because Rett syndrome in mice is reversible, our study is the first step towards a potential cure for this CNS disease.
Watch the webinar "Repairing the Underlying Cause of Rett Syndrome Through RNA Editing" on YouTube