Gene therapy for rare diseases

Gene therapy is a form of therapy in which healthy DNA is given to the patient to repair the error in the patient's DNA. Healthy DNA can be administered in different ways. LentiCure applies lentiviral gene therapy. This therapy uses so-called lentivral vectors: inactivated viruses that have been modified to deliver the healthy DNA in a safe way. This form of therapy is suitable for diseases in which there are errors in the DNA, the genetic diseases.