Glossary

Gene therapy is a treatment method designed to address genetic abnormalities or diseases by introducing or modifying genes within a patient’s body. The purpose is to restore the function of abnormal genes to normal or introduce new functions to enhance the effectiveness of treatment. Its applications have advanced in areas such as cancer and genetic diseases, garnering attention as part of personalized medicine.

Ex vivo gene therapy is a treatment method where a patient’s cells are genetically modified outside the body and then reintroduced into the patient. Initially, cells (typically immune cells or stem cells) are collected from the patient, and the desired genes are introduced, modifying the cells to exhibit specific functions or anti-cancer effects. Subsequently, the modified cells are expanded and retransplanted into the patient. Ex vivo gene therapy contributes to the development of personalized treatments for cancer and genetic abnormalities, mitigating immune compatibility issues by using the patient’s own cells.

The blood-brain barrier is a crucial barrier that protects the central nervous system (brain and spinal cord) from circulating blood. Formed by specialized endothelial cells, this barrier prevents harmful substances and microorganisms from entering the brain. Simultaneously, it allows essential nutrients and oxygen to pass through, maintaining a stable environment within the brain. The existence of the blood-brain barrier is significant from the perspective of brain protection, and understanding it has progressed in neuroscience and research on brain disorders. Since the blood-brain barrier poses constraints on the delivery of drugs to the brain, the fields of medicine and biotechnology are currently focusing on the development of new methods and treatments to overcome this barrier.

Genome editing is a technique that uses technologies such as CRISPR-Cas9 to modify the genes of organisms, bringing innovation to genetic research and the development of therapeutic approaches. This allows for the correction of genetic mutations that cause diseases, raising hopes for treatments of challenging conditions. The advantages of genome editing lie in its ability to correct defects in genes and mutations that cause diseases, restoring normal gene function. As a result, advancements in gene therapy and therapeutic drug development are occurring, opening new prospects for the treatment of challenging diseases.

Suicide gene therapy is an innovative treatment targeting cancer cells. Through this approach, genes specifically introduced into cancer cells are activated, inducing the self-destruction of these cancer cells. This process minimally affects surrounding normal cells, leading to improved treatment precision. Suicide gene therapy is anticipated to be effective, selective, and highly safe, making it a potential widely utilized new approach in cancer treatment in the future.

Neural stem cells are cells with the potential to differentiate into various cells of the nervous system. These stem cells exist in an undifferentiated state and can differentiate into neurons, glial cells, and others when damage occurs to the brain or nervous tissue. Neural stem cells play a crucial role in maintaining and repairing the function and structure of the nervous system, drawing particular attention in the treatment of injuries to the brain or spinal cord and neurodegenerative diseases. Research involving the utilization of these cells contributes to advancements in neural regenerative medicine and the development of treatment methods for neurological disorders.

Pluripotent stem cells are cells with the capability to differentiate into various cell types within the body. These undifferentiated stem cells can transform into cells of different tissues and organs, such as the heart, brain, and muscles, as needed. Due to this characteristic, they are also referred to as “multipotent cells” and have garnered attention in the fields of medicine and regenerative medicine. The application of pluripotent stem cells in regenerative medicine has seen advancements, particularly in the utilization of induced pluripotent stem cells (iPSCs) and embryonic stem cells (ES cells).

Chemokines are proteins involved in immune responses and inflammatory reactions, attracting specific cells to particular locations. They regulate interactions between cells and guide immune cells to sites of infection or damage. Chemokines are released in the body and bind to receptors on cells, transmitting signals to them. Precise regulation is crucial, as abnormal expression may be implicated in the progression of inflammatory diseases and cancer.

A prodrug is a precursor drug that undergoes enzymatic or chemical reactions in the body to be converted into an active pharmaceutical compound. Prodrugs are typically designed to enhance stability, facilitate oral administration, and reduce side effects. Through conversion in the body, they achieve effective therapeutic outcomes.

The CD-UPRT fusion gene refers to a genetic structure in which the Cytosine Deaminase (CD) gene is fused with the Uracil Phosphoribosyltransferase (UPRT) gene. The CD gene codes for an enzyme that converts cytosine, while the UPRT gene codes for an enzyme that converts uracil into uracil mononucleotide. This fusion gene is utilized in research, especially in cancer treatment, and is notable for its prodrug activation activity.
The CD component converts cytosine into a toxic metabolite, and the UPRT component converts it into a usable active metabolite. Within cancer cells, these enzymes cooperate to activate and form complementary metabolic pathways. In therapy, a specific prodrug is administered, and CD-UPRT is activated within cancer cells, inducing selective toxicity against cancer cells. This approach is being researched as an innovative method for cancer treatment, holding the potential for the development of effective and safe therapies targeting cancer cells.