Editing out Disease
Gene editing therapy is a relatively new technology that holds great promise for the treatment of many diseases. Gene editing therapy allows scientists to modify a person's DNA to fix genetic defects that cause diseases. I would like to bring up the discussion about the kinds of diseases that can be cured with gene editing therapy, the current status of gene editing therapy in the developed world, and how to make this technology useful and sustainable in developing countries like Nepal.
Gene editing therapy has proven its ability to treat a variety of diseases caused by genetic mutations, including genetic disorders, certain types of cancer, and viral infections.
Hereditary disorders are caused by genetic mutations passed from parents to children. Gene editing therapy can be used to correct these mutations and prevent the transmission of such diseases to future generations. Some examples of inherited disorders that can be cured with gene editing therapy are:
Sickle cell anemia causes blood vessels to clog as the red blood cells change shape and form a sickle-like shape. Gene editing therapy can prevent changes in blood cell shape that cause sickle cell anemia. This disease is grossly found in the Tharu community living in the western region of Nepal.
Similarly, in the human blood circulatory system, beta thalassemia is a genetic disorder in which there is a decrease in the production of oxygen-carrying (protein) components within hemoglobin. Also, gene editing therapy can be used to correct the mutation that causes beta thalassemia and cure the disease.
Even in cystic fibrosis, a defective gene affects the cells of the lungs and digestive system, leading to frequent infections. Gene editing therapy could be a game changer in this case.
The fact that some types of cancer can even be treated with gene editing therapy is being established. In cancer, cells divide and grow uncontrollably and often spread to other parts of the body. Here are some examples of conditions where gene editing therapy can modify cancer cells and make them less vulnerable by avoiding chemotherapy and other invasive treatments:
Leukemia affects the blood and bone marrow. Gene editing therapy can modify immune cells that identify leukemia cells. Similarly, it has been said that gene editing can cure melanoma, a type of skin cancer.
The therapy can also be used to treat viral infections, such as HIV/AIDS. In viral infections, the virus inserts its genetic material into the patient's cells, causing the cells to produce more virus. Gene editing therapy can be used to modify the patient's own cells to prevent the virus from replicating.
Some other additional diseases that are currently being targeted by gene editing therapies include: Duchene muscular dystrophy; Huntington's disease; Hemophilia
Current Status of Gene Editing Therapy in the Developed World:
Gene editing therapy has made significant progress in the developed world, especially in countries like the United States and China. In the United States, the FDA has already approved gene editing therapy for the treatment of certain types of cancer. In China, gene editing therapy has been approved for the treatment of a rare form of lung cancer.
Despite these successes, there is no doubt that gene editing therapy is still in its early stages. Safety and efficacy need to be established before its widespread use.
Scientists need to develop precise and reliable gene editing techniques. Precision is of great importance to avoid unintended consequences due to the activation of silent genes.
There is a need to develop safe and efficient delivery methods that can only transduce target cells. During the initial Covid Pandemic, we heard about a technique called CRISPR that could only recognize the SARS 2 virus of a specific genotype.
Here we are talking about the technology of altering human genes so it is a serious ethically sensitive topic that requires full evidence assurance that gene editing therapy is used in a responsible and ethical manner with proper guidelines and transparency.
A very important aspect is the assessment of long-term effects. As a new technology, we are still not fully aware of the long-term effects of such therapy, so rigorous studies are expected in the coming days to reliably predict long-term safety and efficacy.
Addressing these challenges will require continued research and collaboration among scientists, regulatory agencies, and the public. With continued progress, gene editing therapy has the potential to revolutionize the treatment of genetic diseases and improve the lives of millions of people worldwide.
Many steps can be taken to make gene editing technology useful and sustainable in developing countries like Nepal, and the first is to invest in gene editing laboratory infrastructure, invest in training, and provide fellowships to indigenous researchers. They should be empowered to collaborate with scientists from the developed world to establish knowledge-sharing platforms. We need to create more education and awareness among people about this treatment method.
The regulatory framework is the weakest link in developing countries including Nepal, we must have a stable law enforcement system to ensure its safety and effectiveness. Poor countries are price sensitive everywhere; however, people prioritize their spending on health services. The government of Nepal should frame a plan to make this technology affordable to all the needy in times of need. Exploring various funding options would be the right approach for any responsible government.
Overall, gene editing therapy can be trusted as a game-changer medical intervention hence developing countries should also participate in taking the necessary steps to make technology useful and sustainable in their respective nations, let us step forward to ensure that its benefits are accessible to all.
I want to dedicate this Article to Mr. Mahabir Pun, founder of The National Innovation Center for his consistent effort to bring national-level focus on Research & Development.