Sylvia Stankov

Mentor: Dr. Barry Byrne
College of Medicine
"I began gaining research experience my freshman year after briefly learning about gene therapy in a biology course. The following semester, I joined a lab investigating that same vector and have since worked in two different labs investigating Adeno-Associated Virus (AAV)-mediated gene therapy. I am passionate about research and continue to be motivated by the intellectual challenge and rigor associated with novel science. Through my immersive lab experiences, I have come to appreciate the impact of biomedical research on the future of medicine, especially the potential of AAV-gene therapy for correction of genetic disorders. Overall, my involvement in research has confirmed my desire to attend graduate school in the biomedical sciences."


Microbiology and Cell Science



Research Interests

  • Gene Therapy
  • Rare Diseases
  • Regenerative Medicine

Academic Awards

  • CLAS Dean's List 2013-2016
  • Anderson Scholar of Highest Distinction 2015
  • Howard Hughes Medical Institute Intramural Research Award 2015
  • University Scholars Program 2016


  • American Society for Microbiology
  • UF Common Reading Program
  • UF Honors Program


  • Saint Kiril and Metodii Bulgarian School
  • UF First Generation Honors Mentor
  • Habitat for Humanity

Hobbies and Interests

  • Reading
  • Traveling
  • Writing and Editing
  • Classical Piano and Flute

Research Description

Testing a Gene Therapy Vector for Prevention and Correction of Cardiac, Molecular Pathology in Pompe Disease
Pompe Disease (PD) arises from mutations in the GAA gene, which encodes the enzyme acid α-glucosidase responsible for the degradation of glycogen in lysosomes. Due to lack of enzyme, glycogen accumulates in lysosomes leading to a cascade of autophagic dysregulation, muscle atrophy, and a decrease in overall muscle function. Although preclinical studies and a current clinical trial of an Adeno-Associated Virus (AAV)-mediated gene therapy treatment are in testing, the effect on autophagy has not been characterized following gene therapy. Understanding autophagy following PD gene therapy is significant in this fatal metabolic disorder arising annually in 1 in 40,000 births. Furthermore, the cardiac hypertrophy and skeletal weakness together lead to cardio-respiratory failure, and without treatment, a life expectancy of one year with increasing disability over time. Currently, the only FDA approved treatment for PD is Enzyme Replacement Therapy (ERT) that relies on a functional autophagic-lysosomal pathway and does not clear the autophagosome accumulation. Investigating PD gene therapy and its effects on autophagy can allow us to move forward in securing an effective treatment for these patients and improving their quality of life. This project aims to evaluate a gene therapy-based approach to prevent and correct autophagic dysregulation in cardiac tissue of the PD mouse model (Gaa-/-). The expectation is that by replacing the defective gene responsible for PD, primary and secondary pathology will be inhibited in the prevention aim, and possibly reversed in the correction aim.