What are genes?
The gene is considered the basic unit of inheritance. Genes are a set of instructions passed down from parents to offspring. They contain the information that determines a person’s specific physical and biological traits. Genes are made of sections of DNA. Every gene consists of four different nucleotide bases, which can be sequenced in different ways.
What are genetic disorders?
Genetic disorders occur when a mutation (a harmful change to a gene) affects your genes or when you have the wrong amount of genetic material.
By receiving half of the genes from each biological parent, there is a possibility of inheriting a gene mutation from one parent or both. Sometimes genes change due to issues within the DNA. This can raise your risk of having a genetic disorder. Some cause symptoms at birth, while others develop over time.
There are three main types of genetic disorders:
- chromosomal: this type affects the structures that hold your genes/DNA within each chromosome. With these conditions, people are missing or have duplicated chromosome material.
- single-gene: these disorders occur from a single gene mutation.
- complex: this group of conditions stems from a combination of gene mutations and other factors, which includes diet, certain medications, alcohol/tobacco use.
How common are genetic disorders?
Around 1 in 50 people are affected by a known single-gene disorder, while around 1 in 263 are affected by a chromosomal disorder. Around 65% of people have some kind of health problem as a result of congenital genetic mutations. There are well over 6,000 known genetic disorders, and new genetic disorders are constantly being described in medical literature.
Due to the constant development of such diseases, the usual medical approach is no longer enough to deal with this problem. For this reason, the need for the development of the field of gene therapy is growing significantly.
What is gene therapy?
Gene engineering is a medical field which primary focus is genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. Simply speaking, gene therapy is a technique that modifies a person’s genes to treat or cure disease.
What are the ways to do this?
Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight it.
Researchers are investigating several ways to do this, including:
- making diseased cells more evident to the immune system. In some cases, the immune system does not fight the diseased cells because it does not recognize them as a threat. In this case, gene therapy allows doctors to train the immune system to better recognize the intruders
- replacing mutated genes. This may help treat certain types of the diseases.
- fixing mutated genes, so that it doesn’t cause the disease
What are the benefits?
In the future, genetic therapies may be used to prevent, treat, or cure certain inherited disorders.
So, gene engineering:
- provides options and opportunities
- prevents the transmission and provides effects passed down through generations
- provides long-lasting effects and has to be given one time in contrast to medication
What are the risks?
Genetic therapies hold promise to treat many diseases, but they are still new approaches to treatment and may have risks, such as:
- unexpected and unwanted immune system reaction, which may cause organ failure
- possibility of causing a tumor
- infection caused by the virus
Is gene therapy worth it?
As far as I’m concerned, the question “is gene therapy worth it?” is wrong as well as the assumption of a yes-or-no answer. This should be an individual and conscious choice of each person. On the one hand, it gives opportunity and hope even in the most difficult cases, however, there is always a risk. When making such a decision, it is worth taking into account all the nuances, because genetic engineering is considered a relatively new and not yet sufficiently reliable direction. The health of each person is individual which means that the approach should be different for everyone.
references:
https://www.medicalnewstoday.com/articles/120574#summary
https://en.wikipedia.org/wiki/Gene_therapy
https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619
https://my.clevelandclinic.org/health/diseases/21751-genetic-disorders
Gene therapy is a new intriguing way of treatment for specific genetic conditions and diseases. I do consider that gene therapy has the potential to cure the symptoms of states that currently have no cure or limited treatment options, such as cancer, inherited genetic problems, and immune system disorders.
But as it is still a new field, this type of treatment has many challenges and risks. It is costly. Additionally, gene therapy is only suitable for specific conditions and may not be effective for all patients. There are also a lot of side effects and risks – for example, negative responses or impact on other genes.
It is essential to carefully consider gene therapy’s potential benefits and risks before deciding if it is the right treatment option for you. It is always a must-do thing to speak with a professional and seek the advice of specialists in gene therapy before making a decision.
I don’t think we should fiddle around with genomes too much. Nature created it as a tool to introduce diversity. Yes, some mutations are bad, but others have incredible benefits. If we just stuck to curing illnesses, it would be great. Unfortunately, what’s likely to occur afterwards is that wealthy people will either want to apply gene therapy to themselves or edit the genome of their children. Over time it would become more and more widespread. At the end, everyone would be the same “perfect” specimen as everyone would have “perfect” genes. I’m not sure it’s really what humans want.