At MPS Matters 2025, Prof Simon Jones from St Mary's Hospital in Manchester talks about research updates for MPS conditions with a particular focus on gene therapies.
Gene addition therapy involves introducing a functional copy of a defective gene to restore its normal activity. This approach can be carried out in two ways: in vivo, where the therapy is delivered directly into the body, or ex vivo, where cells are modified outside the body and then reintroduced.
