Biotech & Medical Device, Biotech Consulting, Client Engagement, Healthcare

AAV Gene Therapy — Structural Biology Research

Peer-reviewed cryo-EM work on the only FDA-approved gene therapy vector

Real engagement, published with the client’s permission.

Cross-Species Permissivity paper as published in the Journal of Virology

Fig. 01 — Journal of Virology, 2022.

2.9 Å Cryo-EM resolution achieved
3 Peer-reviewed publications
+25% Protein purification yield gain

Overview

Structural biology research on adeno-associated virus and its human cell entry receptor, conducted in the Chapman Lab at the University of Missouri and published across three peer-reviewed outputs. AAV is the only gene therapy vector class approved by the FDA — the basis of Zolgensma, Luxturna and Hemgenix.

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

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