DrMawj Muhanad Naseem Adam

Dr Mawj Adam is a Research Officer in the Gene Therapy Research Unit at the Children's Medical Research Institute (CMRI) and an affiliated researcher with The University of Sydney. Her research focuses on developing novel genetic and epigenetic therapies for rare inherited disorders, with particular expertise in AAV gene therapy, genome and epigenome editing, nucleic acid delivery technologies, antibody engineering, X-chromosome inactivation and metabolic liver disorders.

 

Her current work aims to develop therapeutic strategies that reactivate healthy endogenous genes silenced by X chromosome inactivation, creating new treatment opportunities for females affected by X-linked genetic disorders. Using ornithine transcarbamylase (OTC) deficiency as an exemplar disease, she is developing programmable epigenome-editing approaches and clinically translatable mRNA-lipid nanoparticle delivery systems to restore expression of therapeutic alleles.

 

Dr Adam's broader interests include functional genomics, rare disease therapeutics, advanced biologic delivery systems, and translational research pathways that accelerate the progression of innovative therapies from laboratory discovery to clinical application. Through collaborations with researchers, clinicians, and consumer groups, she is committed to advancing precision therapies for patients living with currently untreatable genetic diseases.