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Essay: Gene Therapy Treatment: From Discovery to Mutations & Disease Cure

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  • Published: 25 February 2023*
  • Last Modified: 22 July 2024
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  • Words: 1,335 (approx)
  • Number of pages: 6 (approx)

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Cancer is “the disease caused by an uncontrolled division of abnormal cells in a part of the body” It may lead to organ failure and death. There are many kinds of cancer that attacks our bodies. Some of the most common types of cancer are breast cancer, prostate cancer, basal cell cancer, melanoma, colon cancer, lung cancer, leukemia, and lymphoma. The second most leading cause of death in the United States in fact is cancer; it has and still does affect millions of people today. There are many different types of treatments for cancer but the type those affected varies. It depends on the kind of cancer you have in order to determine your treatment. A common known treatment is radiation. A new treatment is believed to be very promising, it is a gene therapy, called T cell therapy. CAR T-cell therapy uses genetically engineered immune cells. With all the different discoveries of treatment for cancer, the future brings us more success gene therapy is effects will be high and will start journey to end cancer

Gene Therapy is a treatment that modifies genes. If the gene is a mutation, inactive, or just not functioning adequately, then that is what is required for a modification to cease that. Any kind of disruption in genes translates into a disease in the body. Gene therapy may add a new gene to replace the defective ones that could lead to curing that diseases. When the treatment was first developed, results were promised to treat diseases like cancer, diabetes, hemophilia, aids, and heart disease. Throughout the history of medicine and from trials and errors of various other treatments, gene therapy is shown to be the most effective and promising treatment to cure disease among humans.

Around the 1960’s-70’s was when the idea of gene therapy surfaced. There was a science paper published by Theodore Friedmann and Richard Roblin, called “Gene therapy for human genetic disease?”. The first trial in the use of gene therapy was conducted on September 14, 1990. Before the trial, tests in 1985 were the peak of this new beginning. In 1985, the National Heart, Lung, and Blood Institute and National cancer institution discovered tissue culture can correct the cells of patients with adenosine deaminase disorder. In 1986, Studies showed how to transfer correct genes safely and successfully. In 1988, white blood cells were used instead of bone marrow cells and that resulted in an increase in the number of correct genes. In 1989, there was a discovery that the TIL cells work more for cancer. Also, humans were approved the safety of using an engineered virus. Then in 1990, the first gene therapy trial was tested on a four-year-old girl. This girl had a disease that caused her to have defenseless white blood cells, it was called adenosine deaminase deficiency. Her white blood cells were taken and replaced by some normal adenosine deaminase cells effectively.

When gene therapy first started, it was sought to only treat single-gene defects. Today its promised to treat many more. Gene Therapy can be eminent by three types of diseases. There is monogenic disorders, polygenic disorders, and infectious diseases. Monogenic disorder is a single gene that is defective and responsible for disease, it is also hereditary. Some examples of it are hemophilia, sickle cell anemia, Huntington’s disease, Parkinson’s disease, hypercholesterolemia, cystic fibrosis, chronic granulomatous disease, fanconi anemia, gaucher disease, etc. Polygenic disorders can vary on one’s lifestyle or environment, it includes more than one gene. Examples of polygenic is diabetes, schizophrenia, Alzheimer’s disease, cancer, and heart disease. The last, infectious disease is self-explanatory, a disease that is caused by infection, that example would be HIV.

The largest disease focused on is cancer. What cancer is and some facts have already been introduced above. What kinds of cancers named will be further explained. The oldest cancer discovered is called, Osteosarcoma. It is a bone cancer, and it was found in early fossils of mummies. The other kinds of cancer, breast cancer, prostate cancer, basal cell cancer, melanoma, colon cancer, lung cancer, leukemia, and lymphoma. Breast cancer is a cancer that forms in the cells of the breasts. Prostate cancer is in a man’s prostate, a gland that produces seminal fluid. Basal cell cancer begins in the basal cell and is a cancer. Melanoma is the most severe type of skin cancer. Colon cancer is a cancer located at the digestive tract’s lower end and in the rectum or colon. Lung cancer is a cancer in the lungs caused by smoking. Leukemia is a cancer of tissues forming blood. Then Lymphoma is a caner located in the lymphatic system. There are many treatments that treat any of these cancers. There is surgery, radiation therapy, chemotherapy, immunotherapy, targeted therapy, hormone therapy, stem cell transplant, and precision medicine. Surgery removes the cancer from the body, radiation therapy uses a high dose of radiation to kill cancer cells and reduce the size of tumors; Chemotherapy is a treatment that uses drugs to kill cancer cells, immunotherapy benefits the immune system in fighting cancer, targeted therapy changes cancer cells that help grow, divide, and spread. Hormone Therapy helps the growth of the breast and prostate cancers to slow down or stop, Stem cell transplants restore the blood-forming stem cells that were previously destroyed by radiation. Precision medicine give a genetic understanding of disease to doctors to assist them in selecting the treatment for the patient.

Gene therapy will cure diseases by transferring genetic materials. That is its promise, also it can transform viruses. DNA and RNA can be divided based on the patient’s genome. DNA sequences are able to be deleted and additions can be made, like correcting it. This capability of detecting the mutations is a major deal for the future. Gametes, embryos, and zygotes also will have the opportunity to be modified. There are two types of gene therapy Somatic gene therapy, which takes the somatic cells and transfers it. That new DNA from the transfers will not affect one’s eggs or sperm. The other is Germline gene therapy which also transfers, but it does to only a section of DNA, specifically where the eggs or sperm are produced. Somatic gene therapy can also be considered as ex vivo and in vivo. Ex vivo is modification of the bodies exterior and then transplanted back inside the body. In vivo, which is the exact opposite of “ex vivo”, the modifications are within the body.

Gene Therapy as a whole, sounds very safe and promising. But even something good may not always be good. This treatment is still new and still being tested. The risks are still there. It isn’t fully promised on its safety yet, but it is still being studied to determine its security. The issues currently noticed are toxicity, inflammation, and cancer. Specific gene therapy products are out now though, those are what has been approved by the FDA at the moment. These products are only available within the research setting. The United States has limited amounts of what is being sold. The costs of Gene Therapy are extremely expensive. In Europe, the costs were close to $1,000,000. It is expected to be close to that price in the United States.

Gene Therapy has it many promises. Along with the promises comes the side effects. Medicine has come so far throughout the years and have only advanced with time. Now that Gene Therapy is new, it will be more familiar in the future. It will also be more available and used in the next few years. With more research and studies being done, the treatment will be safer in due time. With this type of treatment coming out, it is only the beginning to curing diseases. This treatment can influence other scientists to work on something new. More and more treatments may start to develop after this. Our future is bright with the many advances in science and technology. Gene therapy is the treatment of the future, but not the only one.

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