I am Dr. Jim Selfridge, a dedicated researcher at The University of Edinburgh. My academic scientific career began in 1988 when I joined The University of Edinburgh in a technical role. I later went on to gain a PhD in Molecular Biology within the same prestigious institute.Much of my career has focused on the design, production and characterization of transgenic mouse models of human inherited disorders as a means of developing therapeutic strategies. After many years of studying DNA repair and cancer I joined the laboratory of Sir Professor Adrian P. Bird where my work became centered on epigenetics and in particular understanding the molecular basis of the neurological disorder Rett Syndrome through the use of increasingly refined transgenic models. I have led the creation of sophisticated transgenic mouse models that faithfully mimic many of the neurological features of Rett Syndrome. These models not only deepen our understanding of the disease mechanisms but also serve as crucial platforms for testing novel therapies. In 2007, I was part of a team that showed, for the first time, that Rett Syndrome may in fact be curable. As a disorder caused by a single gene, Rett Syndrome was an ideal candidate for developing gene therapy strategies. Passionate about translating scientific discoveries into tangible solutions and in order to pursue this goal I recently moved to the laboratory of Prof. Stuart Cobb where I am actively involved in developing gene therapies towards Rett Syndrome and other neurological disorders with as yet unmet needs. As part of a research collaboration between The University of Edinburgh and Neurogene this drive resulted in approval for clinical trials that began in 2023.Through innovative approaches and the development of targeted interventions, I strive to improve the lives of those affected.