Most people with type 2 diabetes must take their medications daily. Some must take daily or weekly shots to manage their blood sugar levels. What if we could cure type 2 diabetes with one shot? With gene therapy, we could treat diabetes with ease. Let’s discuss gene therapy, its application to type 2 diabetes, and how patients can benefit from it.
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Persons with type 2 diabetes need to take medications daily or weekly to manage their blood sugar levels.
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Introducing an artificial gene in the pancreas to produce GLP-1 hormone may cure type 2 diabetes.
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The proposed gene therapy has shown promise in animal models of diabetes.
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Human trials begin next year, and researchers are hopeful that gene therapy will be effective.
Diabetes is a chronic disease characterized by impaired insulin production and, thereby, increased blood sugar levels. In the general population, clinicians treat three types of diabetes — type 1, type 2, and gestational. Gestational diabetes occurs during pregnancy when the pancreas does not produce enough insulin. In type 1 diabetes, the pancreas does not produce insulin or produces a negligible amount. In type 2 diabetes, the pancreas produces insulin, but the amount is insufficient for controlling blood sugar levels.
Type 2 diabetes is the most common type, and this article discusses a novel treatment.
Managing diabetes
Since there is no permanent cure for diabetes yet, persons with diabetes need to conduct various self-care practices. These self-care practices include diet, exercise, and, most importantly, medication. Estimates suggest that approximately 88% of persons with diabetes must take daily oral medications, insulin, or both.
Diabetes is a complex metabolic disease. Therefore, if blood sugar is left uncontrolled, it affects other organs. For instance, persons with diabetes are more likely to experience increased levels of blood cholesterol and high blood pressure. Hence, it is crucial that patients follow their regular self-care and medication routines.
However, routine self-care is not always
possible.
What makes self-care difficult?
People with diabetes may experience certain barriers to self-care. For example, people working long hours may not have enough time to exercise or plan their diet. People may also live in food deserts — areas where healthy food is not easily obtainable. Additionally, people from underserved communities may not receive culturally appropriate dietary guidance. Furthermore, unfortunately, insulin is expensive, and uninsured persons with diabetes in the U.S. may not be able to afford to buy insulin regularly.
Given these common challenges, scientists are working to find a permanent cure for diabetes. In some cases, transplanting the pancreas or certain types of pancreatic cells proved beneficial. However, it is difficult to obtain a human pancreas for transplant purposes. Therefore, researchers have explored various possible treatments to induce a patient’s pancreas to produce insulin.
Gene therapy shows promise
In our cells, genes contain instructions to produce various proteins, and these proteins, in turn, make various hormones. If a particular gene is faulty, then the cell cannot produce that specific hormone. However, in gene therapy, scientists focus on disabling or replacing a faulty gene or adding a new gene that functions properly. Harmless viruses are used as a vehicle to introduce this new gene. In the coming years, treatment for chronic diseases such as cancer, cardiovascular diseases, and cystic fibrosis may be possible using gene therapy.
Gene therapy shows promise in treating diabetes and can be implemented for three different goals.
- Target faulty genes that impair glucose absorption in the body.
- Target genes that prevent diabetic complications — such as diabetic neuropathy and diabetic cardiomyopathy.
- Gene therapy can replace these faulty genes and prevent complications.
The third type of gene therapy targets genes that enhance insulin secretion and activity. The beta cells in the pancreas synthesize, store, and release insulin when necessary. These cells become dysfunctional, which leads to type 2 diabetes. Glucagon-like peptide-1 (GLP-1) is a gene that helps beta cells in the pancreas survive longer and improves insulin secretion. In 2022, researchers observed that when the GLP-1 gene was introduced in animal models (mice and pig models), it reduced blood sugar and body weight. Researchers hope that similar results can be replicated in humans. They also hope that one shot of gene GLP-1 and carrier virus will someday cure type 2 diabetes.
Challenges implementing gene therapy
Although gene therapy has shown promising results in animal models, human clinical trials have not yet been conducted. Human trials will likely begin early next year and continue for five to ten years. Gene therapies can be expensive. At present, gene therapy is available for hemophilia B, a blood-clotting disorder. However, it costs approximately $3 million to treat a patient with hemophilia B. Estimates suggest that gene therapy for diabetes may be as costly.
Treating diabetes with novel treatments such as one-shot gene therapy may become a reality in the coming decades. However, until then, persons with diabetes need to manage their blood glucose levels by standard self-care. This self-care may include daily or weekly medications. These oral medications or weekly injections help achieve normal blood sugar levels and prevent further complications. Talk to a doctor to discuss the best-suited medications and lifestyle changes, including exercise and diet.
- Diabetes in America. CHAPTER 39 Medication Use and Self-Care Practices in Persons With Diabetes.
- Biomedicines. Current Advances in the Management of Diabetes Mellitus.
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