List of the Disadvantages of Gene Therapy
- There could be unwanted immune system reactions. The body’s immune system might see the various viruses that we use to replace unwanted genes as invaders that must be extinguished ...
- Current gene therapy methods can sometimes target the wrong cells. ...
- The delivery viruses might recover their ability to create disease. ...
What are the pros and cons of gene therapy?
Pros Of Genetic Therapy
- Offers Hope. There are multiple births that suffer abnormalities and genetic disorders, even with rigorous tests in place for parents.
- Gives Long-lasting and Timeless Effect. There are small prospects of remission when replacing a dysfunctional gene with a functional gene in a condition such as cystic fibrosis, and this ...
- Treats Genetic Disorders. ...
How is gene therapy changing lives?
Gene replacement therapy. It’s a game changer when it comes to treating life-threatening illnesses. It can replace disease-causing genes with healthy genes, knock out a gene that’s not working right, or add a new gene to the body to help fight disease.
How does gene therapy affect a cell?
How does gene therapy work? Gene therapy works by altering the genetic code to recover the functions of critical proteins. Proteins are the workhorses of the cell and the structural basis of the body’s tissues. The instructions for making proteins are carried in a person’s genetic code, and variants (or mutations) in this code can impact ...
Is gene therapy a good thing?
Gene therapy is also a good technique for diseases not researched yet. All of us carry defected genes and may not know it. Gene therapy is a 'medicine" for the future since it can control or eliminate hereditary diseases. The consequences of gene therapy are numerous at this time. As with anything not researched there are critics.
How can gene therapy help?
Advances in gene therapy could help to correct these issues instead of forcing parents into a heartbreaking scenario . 2. Gene therapy could change the perspectives that people have about disease. Roughly 10% of all Americans are affected by a rare disease or condition on any given day.
Why is gene therapy important?
7. Gene therapy allows us to treat the “untreatable” diseases. Gene therapy is potential miracle worker when we start to look at its full potential for humanity. It offers us the opportunity to eliminate, and then prevent hereditary diseases like hemophilia and cystic fibrosis.
What happens when blood stem cells are removed from patients?
When the blood stem cells are removed from patients, retroviruses then deliver working copies of the defective genes to the body. For the gene therapy options which have been approved for use, there are many success stories to consider. Sebastian Misztal is one such story.
Why did Jesse Gelsinger die?
And Jolee wasn’t the only story. A teenager named Jesse Gelsinger also died because of treatments offered inn a clinical research study . We must remember that there are sad stories to tell in addition to the happy ones when evaluating this treatment option.
What is the promise of gene therapy?
The promise of gene therapy is that it can reduce or eliminate the pain and discomfort that these abnormalities cause. 80% of the diseases that we know impact human health in negative ways have a genetic foundation. If we can replace the cells or chromosomes that are at-fault, then it becomes possible to offer relief. 3.
What is gene therapy?
Gene therapy is a medically-based practice that uses normalized genetics to replace genes which are either not present or abnormal for some individuals. Doctors would take the specific gene sequences that need adjustment, and then insert them into the cellular information of the patient in various ways.
Where are gene therapy trials based?
Roughly 70% of the currently active gene therapy clinical trials are based in the United States. Europe approved their first treatment in this area in 2012. These are the pros and cons of this scientific approach to consider.
Why is gene therapy so difficult?
Due to the rapid division of many cells, it is very difficult for the gene therapy to show any helpful results. Therefore patients, who are treated with gene therapy, should be treated with repeated courses of gene therapy. Viruses are used as vectors in gene therapy.
Why is gene therapy not common?
May be the therapeutic genes which are inserted in the body not accepted by the immune system and viral vectors are not useful because they can trigger their pathogenic features in the body...
Why is gene therapy short lived?
It is necessary that when the gene therapy starts functioning, the therapeutic DNA must be inserted into the cells and should start its function. Cells should be long living so that the scientists could achieve their goal.
Why are multiple defects less likely to be treated with gene therapy?
Defect in the multiple genes have less chances that they can be treated with gene therapy because, if vectors are introduced for the treatment of one gene, the other defected genes will remain in the diseased form and disease will not be cured. Short lived nature of gene therapy:-.
How does gene therapy work?
If the disease is caused in the body by mutation in the single gene, then gene therapy can act against these genes effectively. It can easily insert the transgenic genes in the vectors and enter them into the body. These vectors will target the diseased genes and will replace them but if the disease is caused due to the mutations in the multiple genes like heart disease, diabetes, high blood pressure, Alzheimer's disease and Arthritis, then it is very difficult to do gene therapy. Defect in the multiple genes have less chances that they can be treated with gene therapy because, if vectors are introduced for the treatment of one gene, the other defected genes will remain in the diseased form and disease will not be cured.
Can the immune system destroy transgenic cells?
It is possible that the immune system along with the antibodies and t-cells may destroy the foreign object. So when any object that is vector which is carrying the transgenic genes, they might be rejected by the immune system. Then it will also affect the gene therapy. There are chances that the immune system does not allow ...
Is gene therapy good for cancer?
Gene therapy is very much beneficent in treating many dangerous diseases like cancer and hepatitis. As in this technique, new genes are inserted in the body which replace the defected genes and make the cell function in a normal way. but sometimes we don't get the required results in any therapy. Like other technologies gene therapy has also some ...
What are the disadvantages of gene therapy?
List of the Disadvantages of Gene Therapy. 1. There could be unwanted immune system reactions. The body’s immune system might see the various viruses that we use to replace unwanted genes as invaders that must be extinguished before they cause harm.
Why is gene therapy important?
It could improve a person’s life in other ways. The focus of gene therapy research is to create new medicines and treatment approaches that can alleviate suffering. Whether it is a curative solution or not is less important than the positive changes that occur for people who need this help.
How does gene therapy work?
It can work in combination with cell therapy techniques. Gene therapy involves the transfer of genetic materials. Cell therapy is the process of transferring cells with relevant function into the patient when that genetic material is missing in the first place.
What is gene therapy?
Gene therapy is an experimental technique in medical science that uses genes to either prevent or treat disease. The goal with this approach is to create a future world where doctors could treat specific disorders by inserting genes into a patient’s cells instead of using surgery, drugs, or other interventions to improve health.
What is the name of the gene therapy for spinal muscular atrophy?
New treatment options are coming out all of the time. In May 2019, the Food and Drug Administration approved the gene therapy medication called Zolgensma. This product is approved to treat spinal muscular atrophy in children who are under the age of 2.
Why do doctors introduce new genetic material into a patient's body?
Doctors could even introduce new genetic material into a patient’s body to help them fight a disease. The advantages and disadvantages of gene therapy show that this promising treatment option is potentially useful for some inherited disorders, cancers, and viral infections.
Is gene therapy permanent?
The medical impact of gene therapy can create permanent results. Once the faulty genetics are replaced by the correct genes, this therapeutic approach earns the advantage of being a long-lasting, sometimes permanent result.
What are the pros and cons of gene therapy?
Gene Therapy Pros and Cons: Gene therapy is involved in the replacement of a defective gene within a cell’s nucleus with the genetically healthy and functional gene. It is also known as ‘ Gene Augmentation ‘. Genes are basically nitrogenous based codes that encode secret information of all of the amino acid formation in the body.
Why is gene therapy important?
More lifesaving options are available with the help of gene therapy now, so more people mean more diversity and perspectives for future research . By saving the lives of people today, gene therapy can provide a stage for many more potential discoveries in the coming era by those who might not survive without this therapy.
What are the pros and cons of gene augmentation?
Here are some of the pros or advantages of gene augmentation therapy: 1. Genetic Disorders Are Treatable. Rare genetic disorders today affect about 10% of the population in general. Over 7000 distinct diseases exist today and out of which almost 80% are those which are caused by malfunctioning genes.
How does gene therapy work?
Gene therapy is mostly based on technology and our ability to identify, modify and alter specific genes. So, advancement in this field can be achieved at a much faster rate, and as practices or quality of such therapies improves, a decrease in the costs of these therapies will be possible. 8. Increases Lifespan.
What is gene therapy?
Gene therapy provides a strong basis of treatment for those individuals who suffer from infertility and opportune them with starting their own biological families. Using an adjusted and modified genetic therapy method called “ CRISPR “, proper alterations to fertility have occurred in specific mice species.
How many babies die from genetic defects?
These congenital defects are a leading cause in deaths of newborn infants probably 1 in 5 fatalities.
Which disease has a genetic basis?
Even age involved disorders like Alzheimer’s disorder, Parkinson’s diseases , and Huntington’s disease, etc. which have a genetic basis can be provided with medications and drugs formulated by gene therapy research work. 3. Beacon of Light.
Why is gene therapy important?
We can apply gene therapy techniques to plants as animals as well. This allows us to treat their genetic conditions, extending lifespans and stabilizing health. Future applications of gene therapy could create a healthier food chain from start to finish. We could even reinforce the immune systems of plants and animals to naturally withstand disease to prevent species endangerment or extinction. In return, food producers could create more profits because they’d have greater yields.
What is gene therapy?
Gene therapy is a medical technique which utilizes specific, normalized genes to replace the genes that are abnormal or not present in an affected individual. The goal of gene therapy is to correct a disorder caused by missing or abnormal genes. Although the processes of gene therapy are still at an experimental stage, ...
How many people are affected by genetic disorders?
Gene therapy would end the stigma of genetic disorders. In the United States, about 1 in 10 people are affected by a rare disease at any given moment. That means about 33 million people are currently dealing with difficult signs and symptoms of a genetic disorder in some way.
How can gene therapy be used?
Gene therapies can be used to treat disease and genetic disorders. It could also be used to create “perfection” in children. Imagine a world where parents could genetically manipulate their children to be smarter or look a specific way. It would create a new social class of “created” people, where access is exclusive to those who have the financial means to complete such a procedure. It could even lead to a society where those “created” beings suppress those that they feel are “beneath” them.
What is the most successful gene therapy?
5. Gene therapies are most successful when donated tissues are involved. Most gene therapy treatments that are successful today still involve donated tissues, such as bone marrow, from people who are a direct match. Some donations can be extremely painful and create lifelong consequences for those involved.
How many clinical trials have been conducted for gene therapy?
Although the processes of gene therapy are still at an experimental stage, the results achieved have been quite encouraging. Since the 1980s, about 2,500 clinical trials involving gene therapy have conducted worldwide.
Where are gene therapy trials based?
Two-thirds of all gene therapy clinical trials are based in the United States , with most targeting specific cancers or diseases. The first gene therapy approved, however, was in the European Union in 2012. Here are the biggest pros and cons of gene therapy to review.
What is the therapeutic intent of gene therapy?
The toxicity of any treatment needs to be evaluated in two contexts: what is the therapeutic intent (palliative or curative) and what are the other available treatment options? The intent of gene therapy is curative and the alternative of a bone marrow transplant anyway carries with it the risk of inducing a malignancy. Furthermore, the options for children with X-linked SCID if they do not have a matched sibling are pretty dismal.
How many gene therapy trials are there?
There are over 600 clinical trials of gene therapy completed, ongoing or pending throughout the world. Almost 2000 patients have been entered into these trials and about 40 have been children with disorders attributable to mutations in single genes (monogenic). Such diseases are rare but are the best candidates for treatment with gene therapy.
Why is it important to keep the trial open?
Keeping the trial open in this way allows a balance to be struck between risk and caution. Perhaps the most important feature of this compromise position is that parents are drawn in closer to decisions that affect the conduct of clinical trials that have a direct relevance to their children's desperate plight. Interestingly, it is reported that advice to the FDA has now changed and it is now more in line with the GTAC's view, which is that these studies should conti-nue to proceed, but with extra caution.
Is retroviral gene insertion a cause of leukaemia?
These observations clearly suggest that retroviral gene insertion may have been the cause of the leukaemia here. However, other factors such as genetic background and preceding viral infection may have also played a role in the development of this child's disease. A press release from the French regulatory authority rather coyly describes this child's condition as a ‘lymphoproliferative disorder’: perhaps acute T-cell leukaemia might be another description of this complication. 3
Is it illogical to put gene therapy trials on hold?
Long-term follow-up clinical data is always going to be a vital component of any risk assessment of this (or any other) gene therapy strategy. It is thus illogical to put such trials ‘on hold’. It would be more consistent to close them; after all suspending patient recruitment for the required time to complete accurate risk assessment (3–5 years) is tantamount to closure. Further laboratory work alone will not give us an answer to the frequency of treatment-induced malignancy in patients treated with retrovirally based gene therapies.
Is insertional mutagenesis a risk in experimental medicine?
In UK trials of gene therapy for inherited disorders that have involved retroviral vectors, patients and relatives have always been specifically warned of the danger of insertional mutagenesis leading to cancer. Similarly, the Fischer trial considered this to be a possibility and warned the families of the patients who were participating.
Is vector mediated insertional mutagenesis a problem?
Only a few studies in animal models of gene therapy have suggested that vector-mediated insertional mutagenesis could be a problem. However, its spectre has always loomed over the field and now it has apparently been made flesh.
Gene Therapy
Science is an ever-growing field, where there are always new things to be discovered. Science is what leads to the progression of our world, and Biology is what leads to our improvement of life itself. Gene Therapy is a form of biotechnology that works by altering our genes and cells.
Cite this Essay
The Positive and Negative Implications of Gene Therapy. (2020, July 22). WritingBros. Retrieved February 12, 2022, from https://writingbros.com/essay-examples/the-positive-and-negative-implications-of-gene-therapy/
List of The Advantages of Gene Therapy
List of The Disadvantages of Gene Therapy
- 1. There could be unwanted immune system reactions. The body’s immune system might see the various viruses that we use to replace unwanted genes as invaders that must be extinguished before they cause harm. When the white blood cells attack the newly introduced genetic material, it is not unusual for a patient to experience health issues like infla...
Verdict of The Advantages and Disadvantages of Gene Therapy
- Before companies can market gene therapy products for use in humans, the item in question must go through testing to determine its safety and effectiveness. The Food and Drug Administration is the area of government responsible for this oversight. Then the results from each study are carefully examined to determine if the risks of the therapy are acceptable when c…