Genes contain your DNA — the code that controls much of your body's form and function, from making you grow taller to regulating your body systems. Genes that don't work properly can cause disease. Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease.
Gene therapies can work by several mechanisms:
- Replacing a disease-causing gene with a healthy copy of the gene
- Inactivating a disease-causing gene that is not functioning properly
- Introducing a new or modified gene into the body to help treat a disease
Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. Some details of such therapies are give here;
Type 1 Diabetes
Gene therapy for type 1 diabetes (T1D) aims to address the autoimmune destruction of insulin-producing beta cells in the pancreas and restore normal insulin production. Strategies include using CRISPR/Cas9 gene editing to modify pancreatic cells, making them more resistant to immune attack or encouraging beta cell regeneration; encapsulating genetically modified beta cells or stem cells in protective capsules to avoid immune rejection; reprogramming the immune system to prevent it from attacking beta cells, such as by promoting regulatory T cells; and directly delivering genes to stimulate insulin production by enhancing existing cells or converting other cells into insulin-secreting ones. These approaches seek to treat or cure T1D by either regenerating beta cells or making the immune system tolerant to them.
Type II Diabetes
Gene therapy for type 2 diabetes (T2D) targets the underlying causes of the disease at the genetic level, offering potential long-term solutions. Key strategies include using gene editing, such as CRISPR, to modify genes involved in insulin resistance, improving the body’s response to insulin; delivering genes to enhance pancreatic beta cell function or increase their number to boost insulin production; targeting genes that regulate glucose metabolism, particularly in the liver, to prevent excessive glucose output; and reducing chronic inflammation, which contributes to insulin resistance. These approaches aim to restore normal glucose metabolism and insulin function in individuals with T2D.
CAR T Cells
CAR T-cell therapy is an advanced immunotherapy that involves genetically modifying a patient's own T cells to better recognize and attack cancer cells. The process consists of collecting T cells, engineering them to express a chimeric antigen receptor (CAR) that targets specific cancer cell proteins (like CD19 in blood cancers), and reinfusing the modified T cells back into the patient. CAR T-cell therapy has shown significant success in treating blood cancers, including B-cell acute lymphoblastic leukemia, non-Hodgkin lymphoma, and chronic lymphocytic leukemia, with potential for application in solid tumors. This therapy offers advantages over traditional treatments by providing high specificity, boosting the immune response, and in some cases, offering long-lasting remission or cures. Ongoing research aims to expand its use, overcome resistance, and develop "off-the-shelf" CAR T cells from healthy donors. Several CAR T therapies, such as Kymriah and Yescarta, have already been approved for clinical use, revolutionizing the treatment of certain cancers.
CAR NK Cells
CAR NK cells (Chimeric Antigen Receptor Natural Killer cells) are an emerging immunotherapy that combines CAR technology with the innate immune properties of natural killer (NK) cells. Unlike CAR T cells, which rely on the adaptive immune system, CAR NK cells offer a faster, more flexible immune response, as NK cells do not require prior activation to target and destroy cancer cells. CAR NK cells are genetically modified to express a chimeric receptor that recognizes specific tumor antigens, such as CD19 in blood cancers. They can be collected from either the patient's own blood or healthy donors, and after expansion, are reinfused to target and kill cancer cells. Advantages include a lower risk of graft-versus-host disease (GVHD), reduced cytokine release syndrome (CRS), and potential "off-the-shelf" availability, making them easier and more affordable to produce. While CAR NK cells have shown promise in hematologic cancers, research is ongoing to extend their use to solid tumors and improve their effectiveness in overcoming immune suppression within tumor environments. With their potential for faster action, reduced toxicity, and broader applicability, CAR NK cells are a promising alternative or complement to CAR T-cell therapy in cancer treatment.
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