Dr Mawj Adam is a Postdoctoral Research Officer in the Gene Therapy Research Unit at Children's Medical Research Institute (CMRI). Her research focuses on developing innovative epigenome editing therapies for rare genetic diseases, with a particular interest in X-linked disorders. By combining gene therapy, functional genomics, epigenetic engineering and precision medicine approaches, she aims to develop therapies capable of restoring healthy gene expression without altering the underlying DNA sequence.
Mawj completed a Bachelor of Medical and Molecular Biosciences (First Class Honours) at the University of Technology Sydney before undertaking her PhD at the University of Sydney under the supervision of Professor Ian Alexander. Her doctoral research developed novel epigenome editing strategies for X-linked liver diseases using adeno-associated viral (AAV) vectors, establishing new approaches for the targeted regulation of gene expression in vivo.
Her current research focuses on developing next-generation epigenome editing platforms for X-linked rare diseases. Using advanced functional genomics, single-cell technologies and epigenome engineering approaches, she investigates how epigenetic mechanisms contribute to disease and how they can be therapeutically manipulated to restore expression of healthy genes. Her work spans therapeutic target discovery, development of programmable epigenome editors, and the advancement of clinically translatable delivery technologies, including lipid nanoparticles (LNPs) for transient delivery of epigenome-editing cargos. Ultimately, her goal is to develop safe, durable and scalable precision medicines that improve outcomes for patients living with rare genetic disorders.
Outside the laboratory, Mawj enjoys drawing, painting and travelling.