Award Recipient: Fateme Babaha, PhD Candidate, Pathology and Molecular Medicine, Queen’s University, Kingston, Ontario
Supervisor: Dr. David Lillicrap
Conference: Norman Bethune Symposium on April 18, 2024
Location: Vancouver, BC
Oral/Poster Presentation Title: Improving AAV-mediated FVIII transgene expression using enhancer sequences from the F8locus
I am delighted to share my experience at the 2024 Norman Bethune Symposium, which was made possible by the generous External Travel Award from the Centre for Blood Research (CBR). This was my first time attending and visiting the University of British Columbia and the beautiful city of Vancouver. During the symposium, I had the opportunity to learn about the latest research findings, innovative treatments, and improvement in patient care within the field of blood research. I really enjoyed networking with experts of the field and junior scientists during poster presentations and the speaker dinner. These events provided me with invaluable insights on my current project and future career path. I also received fantastic suggestions for exploring the city!
At the symposium, I presented a poster and gave a talk entitled “Improving Adeno-Associated Virus (AAV)-mediated FVIII transgene expression using enhancer sequences from the F8 locus”. This is one of my ongoing PhD projects at the Department of Pathology and Molecular Medicine at Queen’s University, under the supervision of Dr. David Lillicrap. This project addresses the decline of FVIII expression following AAV gene therapy in hemophilia A patients. We aim to achieve increased and persistent transgene expression by employing a hyper-active element from F8 locus (FVIII-Padua) in the AAV expression cassette. We have confirmed that once inserted in a reporter plasmid, this regulatory element is able to significantly increase downstream gene expression in different cell lines and mice. We are also interested in factors affecting vector uptake and its persistent in the nucleus and epigenetic remodeling of the transgene as a potential explanatory molecular mechanism responsible for the decline in FVIII expression. Through this six-minute oral talk, I practiced the skill of effective science communication, learning how to present the study, its objectives and results concisely yet informatively.
I am very happy that I came across CBR’s LinkedIn post about this symposium and am grateful to them for funding my attendance and giving me the opportunity to share my research findings. The event’s organization, the diversity of talks, and networking opportunities were all great. In the end, I felt like I found my science community and I am very much looking forward to reuniting with them at future CBR events.
*With funding from Canadian Blood Services & the Sheldon Naiman and Linda Vickars Endowment Fund, the CBR supports travel and accommodation expenses for trainees to attend and present at the EWD symposium.