Gene and Cell Therapy

New hope in treatment

Gene and cell therapy target the very root cause of a disease, either at the level of the affected gene or the cells themselves.

Gene therapy is a modern form of treatment that addresses diseases caused by the malfunctioning of a gene. The advantage of this therapy is the potential to completely cure the genetic ailment, unlike traditional treatments that only alleviate symptoms. With gene therapy, a normal gene is inserted into the patient’s cell, replacing the damaged gene responsible for a particular disease. Gene therapy strategies fall into two main categories:

  1. Ex vivo – Specific cells are removed from the patient, genetically modified in a laboratory, and then returned to the patient.
  2. In vivo – The correct gene is directly delivered to a cell inside the patient using a special carrier, e.g., nanoparticles. This carrier must be safe not only for the patient but also for the environment and the professionals handling it. 

Gene therapy is currently one of the most dynamic fields in biotechnology, both due to its rapid advancements and the emergence of new possibilities. The development of new carriers, influencing the immune system, and precise manipulation of the human genome were unimaginable a decade ago. However, now they are opening new areas of medical application.

For instance, gene therapy is used to treat Spinal Muscular Atrophy. The drug Zolgensma works by creating a new copy of the gene that encodes the necessary SMN protein. In 2019, it was approved for treating children under two years of age with this disease. This has led to the inclusion of Spinal Muscular Atrophy in newborn screening.

Cell therapy is custom-developed for a specific patient, often using their own biological material. The aim of cell therapy is to restore or modify a certain group of cells in our body so that it can independently combat the disease. It can utilize the patient’s cells or those of a donor, like bone marrow transplantation for leukemia. For example, CAR-T cell therapy is a targeted treatment that leverages the anti-tumor effect of a patient’s own immune system. In the lab, the patient’s white blood cells are modified to recognize and destroy cancer cells. Producing these drugs takes several weeks. Currently, CAR-T therapy is designed for patients with certain types of lymphomas and leukemias, where traditional treatment has failed. Cell therapy is still considered experimental. Scientists are diligently working to make it commonly available to all oncology patients over time. These „living“ drugs are also administered in the Czech Republic and, in selected cases, are covered by public health insurance.

Supplementary Materials