Gene therapy and cell therapy: a breakthrough in the medicine of the future

In the world of modern medicine, we are on the verge of a real revolution. If a decade ago, genetic editing and cell cultivation seemed like fantasy, today they are becoming a reality — not only in laboratories, but also in clinics. Gene therapy and cell therapy are two interconnected areas that are opening a new era of treatment for diseases that were previously considered incurable.

🔬 What is gene therapy?

Gene therapy is a treatment method in which genetic material is introduced or altered into human cells to correct or replace defective genes.

Simply put, instead of fighting symptoms, doctors work with the cause — at the DNA level.

Modern technologies such as CRISPR-Cas9 allow us to “edit” genes with high precision, correcting mutations that cause hereditary diseases: muscular dystrophy, hemophilia, sickle cell anemia, etc.

Real cases:

• In 2024, the FDA approved the first CRISPR therapy for sickle cell anemia (from Vertex Pharmaceuticals and CRISPR Therapeutics). This was a historic breakthrough that proved that gene editing is safe and effective.
• In Ukraine, research is being conducted using genetic methods in the treatment of cancer, in particular, a program to study genetic markers for the selection of personalized therapy is underway at the National Cancer Institute.

🧫 What is cell therapy?

Cell therapy is a method in which living cells are injected into the body to repair or replace damaged tissue. This can be:

• Stem cells, which are capable of turning into any type of cell — from nerve to heart.
• Immune cells modified to fight cancer (CAR-T therapy).

Real cases:

• In the US, CAR-T therapies have already saved thousands of leukemia patients where traditional chemotherapy was powerless.
• In Ukraine, in 2025, the Oberig Clinic in Kyiv began preparations for clinical trials using mesenchymal stem cells to repair tissues after burns and injuries.
• In Japan, Professor Takahashi’s team (RIKEN) was the first to use cells derived from induced pluripotent stem cells (iPS) to treat retinal degeneration — and achieved vision stabilization in patients.

⚙️ How it works

1. Material selection – cells or genes are taken from a patient or donor.
2. Modification – in the laboratory, cells change or activate the desired genes.
3. Administration into the body – prepared cells or vectors are delivered to the patient.
4. Monitoring and adaptation – doctors monitor how the body responds and how tissue function changes.

🌍 Why is this important?

• Treatment of hopeless cases. For diseases that are not treatable with drugs (such as spinal muscular atrophy), gene therapy is the only chance.
• Minimal side effects. Instead of systemic chemistry or radiation, the effect is spot-on.
• Personalization. Therapies are individually tailored to the patient’s genetic profile.
• Long-lasting effect. One course can replace years of treatment.

🚧 Challenges

• High cost (from $100 thousand per course).
• Ethical issues of gene editing.
• The need for strict security controls.
• The availability of such technologies in developing countries is limited.

🔭 The Future: Synergy of Science and Technology

Today, science is moving towards combining gene therapy, cell therapy, and artificial intelligence.

AI helps predict which changes to the genome will have the best effect, and 3D bioprinting allows you to create tissues and even organs from a patient’s cells.

Thanks to international research and the participation of Ukrainian scientists, these technologies are gradually moving from experiments to clinical practice.

📚 Sources for further reading:

• World Health Organization – Gene Therapy Advances (2025)
• Nature Medicine – Gene Editing in Clinical Use (2025)
• CRISPR Therapeutics – Clinical Pipeline
• Kyoto University iPS Cell Research
• National Cancer Institute of Ukraine – Genetic Research