Principles of gene therapy
DNA is a kind of “instruction manual” for the organism. It contains encoded information about the structure and functioning of cells, organs, tissues and biological processes. If there is an error in the genes, the cell does not work properly. This causes serious diseases, from muscle atrophy to cancer.
- Gene therapy corrects such errors. For this, researchers use several technologies:
- Replacing a damaged gene by introducing a “corrected version” into cells.
- If a gene becomes too active due to a mutation, that section of DNA is switched off.
- Activation of the “dormant” gene allows the use of reserve DNA regions. They are able to replace the damaged ones, but remain inactive without an external signal.
- Genes are introduced into cells using special carriers. Most often, modified viruses are used that can penetrate into a cell and embed new information into its DNA. However, the virus does not cause harm to the body.
Diseases that can be cured
Researchers have already developed methods for treating several serious diseases. Among such diseases, there are:
- Spinal muscular atrophy (SMA). This disease affects the nerve cells that control muscle movement. Without treatment, the muscles gradually weaken and the person loses the ability to walk, breathe, and swallow. The drug Zolgensma corrects the genetic defect and restores the functioning of the nervous system.
- Haemophilia. People with this disease have trouble with their blood clotting, and even minor injury can cause severe bleeding. Gene therapy helps the body produce the proteins needed for normal blood clotting.
- Hereditary diseases of the retina. Some forms of blindness are linked to mutations in DNA. Doctors have learned to replace damaged genes to restore vision.
- Cancer. Gene technologies help the immune system recognise and destroy cancer cells. Methods already exist and are used in practice that significantly increase the chances of recovery.
- Researchers claim that the gene therapy could one day treat diabetes, heart disease, neurodegenerative disorders and many other conditions.
Development in Russia
Russian researchers are actively developing new treatment methods related to DNA modification. There are already several successful projects in the country.
- In 2021, the drug Zolgensma was registered in Russia. This is the first gene therapy against SMA that is administered once. Russian scientists have developed their own analogue. It is undergoing clinical trials and some patients have already received the therapy.
- Biocad is developing a drug that will allow patients to avoid regular injections of blood clotting factors. Tests are taking place in 11 cities, including Moscow and St. Petersburg.
- In the laboratories of NTU Sirius, scientists are working on a method that will help restore vision to people with retinitis pigmentosa and Leber’s amaurosis. Researchers are developing a technology that can replace damaged genes and stop the destruction of retinal cells.
- At Sechenov University, genetic methods for fighting cancer are being developed. Scientists are working on a therapy that can activate immunity against tumour cells.
The Russian legislation regulates the conduct of clinical trials and introduction of new technologies, thereby ensuring the safety of patients. The MARUS company, which organises trips to Russia for medical purposes to treat complex diseases, also operates in accordance with the requirements of the law.
Gene therapy opens the way to treating previously incurable diseases. Russia is actively participating in this process and creating its own innovative developments. In the coming years, patients will have new chances for recovery, and medicine will reach a new level.