Gene therapy
In gene therapy, genetic material is introduced into cells or specifically modified to treat or prevent diseases.
Detailed description
Gene therapies can supplement, replace, inactivate, or specifically modify genes. To do this, for example, functional genes or genome-editing tools can be introduced into the target cells.
The treatment can be performed outside the body (ex vivo): In this process, cells are harvested, genetically modified, and then reintroduced. In in vivo treatment, the therapeutic components are delivered directly into the relevant tissue. Among the transport vehicles that can be used are modified viruses, known as viral vectors.
Gene therapies are already being used today to treat various diseases. Additional gene therapy approaches are the subject of intensive research and clinical trials.
