Every day, scientists discover and introduce new and improved treatment methods for many dangerous and life-threatening health conditions, including cancer treatments. One of the latest and most promising treatments is gene therapy, which can potentially change and save many lives in 2023. Gene therapy is a particular method of treatment that, in simple words, can correct, alter, or replace the body’s genes that cause a disorder or disease such as cancer. It has different types and is effective, with a high success rate for inherited or acquired diseases and disorders. This explains why gene therapy in Oman can revolutionize its healthcare system and help many Omanis end their pain and suffering with a safe treatment method. To begin acquiring a better understanding of gene therapy in Oman, it is essential first to spread awareness about this treatment’s uses, potential risks, eligible candidates, different types, etc. This article will cover these different aspects and provide some of the most important information everyone should know about gene therapy.
Gene Therapy
In simple terms, a gene is the basic physical component of heredity that stores important information essential to form human function and appearance, such as hair and skin type, which will also be transferred from one person or generation to the next. However, since the human body contains hundreds of these genes, the function of a gene is more complex than this. Therefore, even if one is missing or malfunctioning, whether by inheriting or acquiring, the person will develop genetic disorders or diseases, such as cancer, which may also cause many other health problems. Gene therapy is one of the treatments that approach adding the missing gene or altering the dysfunctional or defective one through a particular process to boost the body’s strength in curing the disorder or disease. Yet, gene therapy in Oman, as a new treatment method for many diseases, has various aspects that Omanis must be aware of before deciding whether to do it.
Uses
Gene therapy can potentially treat various diseases and is the best option to treat them once for the rest of a patient’s life. Gene therapy in Oman can help many patients to repair a mutation and treat their genetic disorders and other diseases, including cancers, severe combined immunodeficiency or SCID, cystic fibrosis, familial hypercholesterolemia, inherited vision loss, cerebral adrenoleukodystrophy, and blood disorders such as hemophilia A and hemophilia B. In addition to conditions that gene therapy can treat, it is a useful option for curing many rare diseases that can only happen to one person among several million others. This means it can be a life-changing experience for them. However, considering the potential, gene therapy or gene editing still needs to be improved in its ability to target more mutations and other conditions due to its sophisticated mechanism and high expenses, which are only affordable to some people.
Mechanism
After discovering some of the most significant applications of gene therapy in Oman, it is important to understand how gene therapy works and its mechanism. Generally, this treatment has different types and branches, all of which scientists have indicated to some particular disease or disorder. However, as mentioned earlier, gene therapy has three main mechanisms: gene editing, addition, and gene replacement therapy. Editing or repairing involves targeting only the section of mRNA (messenger ribonucleic acid) that contains the mutation instead of replacing the entire gene. Gene addition is the approach to inserting a functional copy of a gene that is whether missing or dysfunctional. And lastly, in gene replacement therapy, a faulty gene is replaced by a new gene, as its name implies.
Whether a patient with a genetic disorder needs one of these gene therapy techniques or other gene therapy methods in Oman, such as gene silencing therapy, they should usually follow certain steps to finish their treatment. However, they are simple and will take little time. First, before proceeding with gene therapy treatment in Oman, patients must determine if this treatment is suitable and understand its pros and cons. After this, with the help of their doctors, patients must choose an approved product to begin the treatment and move on to the third stage, which is doing the one-time treatment. And finally, patients can recover at home and may need to schedule some follow-up appointments.
Types
- In vivo gene therapy: Gene therapy in Oman has two main or general types, and the first type is in vivo gene therapy. Simply put, in vivo delivers the healthy copy of the genes to the patient’s cell through viruses or special products that scientists have made particularly for this treatment.
- Ex vivo gene therapy: Gene therapy in Oman has another main type: ex vivo. This technique combines gene and cell therapy; it needs the removal of a particular cell to add or alter a healthy gene and then transplant the cell into the patient.
Potential Risks
Since gene therapy can be a sophisticated process, especially when the patient needs to put a virus in their body in vivo, many patients are concerned about its safety and potential risks. Before jumping to any conclusion, they must consider that every gene therapy type and product in Oman has undergone vigorous testing and approval. In addition, the viruses in gene therapy products are specifically manipulated to ensure that they do not threaten the patient, much like the vaccines. However, before proceeding to do gene therapy in Oman, patients need to consider the following potential risks:
- In 2023, gene therapy in Oman has the potential for significant progress, and the FDA has approved many related products. Despite this, there may still be unknown long-term side effects.
- The immune system might mistake the virus or viral vector for a potential threat, causing fever, inflammation, and tiredness by reacting to it.
- There is also the possibility that the viral vector could lead to a new mutation by inserting the genetic material into the wrong DNA stretch.
The Impact of Gene Therapy on Oman’s Healthcare System
Gene therapy in Oman not only helps patients fight common or rare diseases that they currently may have, but it also has the potential to eradicate many other diseases nationally and globally. Recent studies have shown that hundreds of millions worldwide have many innocent children and rare diseases and genetic disorders. Gene therapy brings hope that it can be the best treatment for many people and help others initially prevent these diseases. Hopefully, It will eliminate many rare genetic disorders, first in Oman and then in the Arab world. However, not all patients are eligible for gene therapy in Oman, and some challenges also limit access to this treatment.
Candidates for Gene Therapy
As mentioned earlier, gene therapy in Oman has yet to be expanded, and research on this treatment continues, most of which is on life-threatening disorders and diseases. So many scientists in Oman, including those at Opal BioPharma, are working on expanding this research on gene therapies. With the hope that more people with rare diseases will be able to experience the best possible treatment and the country will be independent in treating them. In addition, the FDA approved around 30 gene therapies, including Roctavian (approved in June 2023), for treating severe hemophilia A in adults. In recent years, it has also approved some other significant gene therapies, including those for hemophilia B, CALD, beta-thalassemia, spinal muscular atrophy, and retinal dystrophy. On the other hand, it is worth noting that gene therapy eligibility does not solely involve approval, but the patient’s health also plays an important role.
Challenges of Oman’s Gene Therapy
Though gene therapy has faced many challenges and barriers initially, the cost is the primary challenge with Oman’s gene therapy in 2023. Gene therapies can be so expensive that only a few patients can afford them. These high expenses also cause other problems; manufacturing is the first. As a result, all of these together will cause limitations and barriers to access, meaning many patients cannot provide gene therapy and benefit from a life-long treatment.
Gene Therapy Benefits
Gene therapy in Oman can be beneficial in various ways for the country and also for many patients with genetic disorders or other diseases. In addition, it can eliminate or prevent many diseases in the country and stop some infections, such as HIV. Some of the most important benefits of gene therapy include:
- Curing rare genetic disorders
- Preventing genetic disorders
- Curing Cancers
- Benefiting the next generations in Oman
- Improving the medical situation in the country
- Long or lifetime treatment
- One-time treatment
- Helping the overall Arab world’s health
- Increasing the hope for a better life
Summary
Gene therapy treats genetic disorders and other diseases, including cancer, hemophilia, CALD, cystic fibrosis, familial hypercholesterolemia, inherited vision loss, etc. It is also known to be the best way to cure many rare diseases that have been causing suffering to hundreds of million people worldwide. Gene therapy in Oman has different types and methods. Gene editing, replacement, and addition are some of the most important ones through which a dysfunctional or faulty gene will be replaced or corrected. It also includes two types: in vivo and ex vivo gene therapy. The first one involves a process in which the new and healthy gene will enter the body with a viral vector, or put; the treatment mainly happens inside the patient’s body. The second one involves a process that needs the removal of the faulty gene and replacing it with a healthy one. Despite being the best way to cure or prevent many rare diseases or genetic disorders, it yet needs to be expanded in Oman, and its cost has been an important challenge for patients. However, there is hope that gene therapy in Oman will improve significantly in 2023, helping many patients to experience lifelong treatment and a healthier life for the whole country.
