Cell and Gene Therapy: Revolutionizing Healthcare A Glimpse into the Promising Future of Medicine
Introduction to Cell and Gene Therapy
Cell therapy involve the modification or manipulation of genes and cells in the human body to treat disease. The basic approaches include replacing missing or mutated genes, introducing new genes, or modifying existing genes. These therapies aim to use the natural healing powers of the body by repairing or replacing genes that cause disease.
History of Cell Therapy Development
The early concept of cell therapy dates back to the 1970s when scientists first discovered that genes could be transferred between cells and organisms. However, it was not until the late 1980s and early 1990s that significant progress was made towards human application. Some of the key early milestones included the first successful gene therapy clinical trials in 1990 for severe combined immunodeficiency, as well as FDA approval of the first gene therapy product, Kymriah, in 2017 for certain types of leukemia and lymphoma. Since then, research has accelerated and many new cell therapy products have entered clinical trials or been approved for other conditions.
How Cell Therapy Works
Cell And Gene Therapy involves introducing new cells into the body to replace or repair damaged tissues or cells. Some examples of cell therapies that have been researched or approved include hematopoietic stem cell transplantation, mesenchymal stem cell therapy, chimeric antigen receptor T-cell (CAR T-cell) therapy, and gene-edited cell therapy. The basic approaches are autologous cell therapy which uses a patient's own cells and allogeneic cell therapy which uses donor cells. Cells can be introduced directly into the body via injection or transplantation into organs or tissues. The new cells then engraft and function similarly to native cells.
How Gene Therapy Works
Gene therapy works by introducing genetic material, usually DNA or RNA, into cells to compensate for abnormal genes or produce a therapeutic effect. Viruses are often used as vectors to deliver gene therapy material directly to cells. Some common vector types include retroviruses, lentiviruses, adenoviruses and adeno-associated viruses. Gene therapy can operate via several mechanisms such as replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating a mutated gene that is causing disease, or introducing a new gene into the body to help fight a disease. Gene therapies are being researched as one-time treatments that could provide a long-lasting effect.
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