Gene Therapy

UniQure is investigating the potential of gene transfer to cure patients with genetic disorders. Viruses are used to deliver normal copies of a gene to cells, enabling the production of functioning proteins and potentially curing genetic disorders such as cystic fibrosis. This new genetic material is not integrated into the DNA and so is not passed onto future generations.
The educational animation developed by Random42 sits on the UniQure website and demonstrates to a wider audience how gene transfer technology functions within the body.