Eimina Dirvelytė, a PhD student at Vilnius University's Life Sciences Center, won a prize in special competitive session at the international conference "Translational Research: Bridging Gaps, Improving Lives," which marked the opening of VU Medical Science Center. The young researcher presented her research idea aimed at developing a therapy for the rare genetic disorder Sanfilippo syndrome and was awarded the top prize from the Future Biomedicine Fund – 2,000 euros.
In this competition dedicated to translational research, participants had three minutes to present their ideas, followed by questions and feedback from an expert panel. Ten research ideas from PhD students and young scientists at Vilnius University were selected for the competition.
Innovative Approach to Sanfilippo Syndrome Treatment
In her presentation, E. Dirvelytė proposed a personalized therapeutic solution for Sanfilippo syndrome, a rare inherited neurodegenerative lysosomal storage disorder that causes severe damage to the nervous system. Currently, there is no effective treatment for this disease, but the young researcher explained that the mutations causing it can be precisely corrected using modern CRISPR-Cas gene-editing tools, specifically prime editing.
In her project, E. Dirvelytė plans to use herpes virus vectors for a single component delivery of gene-editing tools into mammalian cells. Initial experiments will be conducted on cells from mice carrying the mutation that causes Sanfilippo syndrome. Later, the therapy's applicability will be studied in more detail using both ex vivo tissue cultures and in vivo mouse models. To translate this approach to human condition, induced pluripotent stem cells derived from patients will be differentiated into neurons, and the patient-specific mutations will be corrected using prime editors.
Scientific Enthusiasm and Recognition
The competition participant expressed joy over her win and recognition, which further motivates her to continue her scientific experiments.
"Since this idea is closely related to my PhD research, the experts' belief in this project and the award encourages me to pursue the research despite the challenges," said the PhD student.
E. Dirvelytė is a second-year biochemistry PhD student involved in other scientific projects as well. She collaborates with research groups both in Lithuania and abroad, frequently presenting her research findings at various international conferences. In addition to her research activities, the PhD student also participates in academic activities, teaching undergraduate students.
The experts attending the conference emphasized that such achievements by young scientists are essential for promoting innovation in the field of biomedicine and ensuring the progress of scientific research in clinical practice.
In addition to Life Sciences Center PhD student Eimina Dirvelytė, Kajus Merkevičius from the VU Faculty of Medicine was also recognized for his presentation "Translating Patient Data into Targeted Therapeutic Strategies in Pyruvate Dehydrogenase (PDHc) Deficiency."
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| Kajus Merkevičius, representative of the Future Biomedicine Fund Dr. Vytautas Naktinis, and Eimina Dirvelytė. Photo: Vilnius University Faculty of Medicine. |