AAV Gene Therapy — Structural Biology Research

The problem

Designing efficient gene therapy vectors depends on understanding how AAV engages its receptor — and on knowing how far that engagement generalises across species, which determines which capsids are viable candidates.

What we did

1. 01Applied cryo-EM to characterise AAV–AAVR interactions across serotypes.
2. 02Used COOT and PyMOL to interpret electron density maps and refine macromolecular structures.
3. 03Solved the structure of goat AAVGo.1 at 2.9 Å and its complex with the human AAVR receptor, establishing cross-species receptor permissivity.
4. 04Optimised protein purification protocols, increasing target protein yield by 25%.

Delivered

• Peer-reviewed publication in Viruses (MDPI)
• Peer-reviewed publication in Journal of Virology (ASM)
• First-author conference research, ASV 2023 and WCSB 2023
• Refined macromolecular structures of AAV and AAVR

Outcome

Three peer-reviewed outputs, presented at national conferences in the United States and featured internationally in Belgium and Italy.