Genes contain your DNA — the code that controls much of your body's form and function, from making you grow taller to regulating your body systems.
The regulated expression of the components of an organisms’ genome creates that organism, our genotypic code controls what we look like today.
Our physical, intellectual and behavioral traits that identify us as individuals are all a product of our genes.
In today's article we are going to talk about gene therapy.
Proteins that are either structurally or metabolically active in living tissue are essentially coded by genes, which are hereditary materials composed of nucleotide sequences.
The biological system that make up an organism is intricately detailed, its assembly and function is a responsibility of the tight regulation of gene expression.
If the regulated expression of gene is balanced, because internal or external influences could distort the regulation; therefore our genes would become faulty and the resulting malaise could become a disease to our bodies.
Proteins can then be malfunctioning or may not be produced at all, by the faulty genes; due to alteration of gene sequences or the regulation of gene expression.
The normal activity of cells is also disrupted, due to the mutated or absent proteins; which would lead to the expression of an illness in the body.
In an attempt to develop therapies the scientific community has thoroughly dissected many diseases, to fix defective gene expression or to interfere with disease mechanisms.
Current treatments often consist of oral drug administration to deliver temporary symptom relief.
Many obstacles such as harmful side effects, drug resistance, cost and addiction associated with long-term use often accompany such therapy.
In the search for more efficient and permanent alternatives, scientists are studying the potential use of viral vectors as a vehicle for gene therapy.
Genes that don't work properly can cause disease that may or may not be curable, so this is where Gene Therapy comes into play.
Many researchers have use this technological advancement to perform research and prove that there are benefits to gene therapy and that it is safe and effective to use.
Gene therapy is essentially the introduction of healthy genes to cells of a patient who has bad genes, which fail to encode a vital protein for proper body functionality.
Health providers have discovered that by using genes, they are able to treat or prevent diseases; therefore, they experiment with genes to find cures for disorder, by inserting a gene into a patient's cells, avoiding the use of drugs or surgery.
The replacement of mutated gene that causes disease with a healthy copy of the gene, inactivating or knocking out a mutated gene that is functioning improperly and introducing a new gene into the body to help fight a disease, are some of the approaches that are being discovered and tested with gene therapy.
The idea behind gene therapy is seductively simple and direct, I mean if a person has a disease that is caused by a faulty gene; replacing that gene, by using a virus as a carrier and then place the virus into the person's tissue, the new gene would provide the genetic code the patient is lacking, for proper cell function.
However, the process of replacing genes in a person's body is not as simple, and it involves a complex process.
Gene therapy involves biochemical problems of gene deliver, gene control and duration of gene action, which are technologically challenges for research doctors who study gene therapy today.
The safety of patients is also a paramount concern in the study of gene therapy; the field is very promising option for the treatment of many diseases.
After years of anticipation, gene therapy has already accomplished plenty; inherited disorder, some types of cancer and certain type of viral infections are being treated right now with the use of gene therapy.
The technological advancement however, remains risky, and it is still under a lot of scrutiny, because safety is the number one concern, and until all the safety precautions have been taken; gene therapy will only be tested on patients who have a disease that is terminal or does not have a cure.
In order to correct defective genes to cure a disease or to help your body better fight diseases, researchers use gene therapy, they investigate several way to correct faulty genes in a patient's body.
Ever since the discovery of DNA, the potential of treating and preventing disease, by modifying and changing the expressions genes are capturing the imagination of many scientists.
Gene therapy as a viable approach for treating a broad spectrum of acquired and inherited disease; although there has been many failed clinical trials in the field.
Gene therapy is a clinical application of modern day molecular biology, and while the concept may seem simple, the translation into effective therapies has been challenging the scientific community.
The revolution of biotechnology and molecular biology, that formed gene therapy, started with a procedure called gene replacement, which simply means that gene therapy is introduced into the cells of patient.
During a therapy of this kind, the added gene does not change the structure of a non-functioning gene.
The Concept of Viruses for The Realization of Gene Therapy
The concept of viruses as a driving vehicle to transport gene to a specified group of cells, in a way that genes would migrate into the nucleus and not cause any other damages the cells; helped scientist overcome the drug deliver technical challenge that they faced.
The DNA that is expressing the new gene, is now available to be transported to its right destination, with less challenges and problems, and the transportation process is secured, save and effective because of the use of viruses as transporter.
When a virus is used to transport modified or functioning genes into a patient’s cell, is called a vector; one of the reason why viruses are used as vector is because of the virus’ ability to infect its host and replicate its DNA, once inside the host.
The introduction of the viral genome into the cell is said to be the phase one of the viral life cycle, which is also called the infection life cycle.
After infection the gene is ready to manufacture virus regulatory products, which are used by the virus to replicate; structural genes are expressed and new viral particles are assembled.
After observing the life of a virus scientists have discovered that the life of a virus and its mechanism can be used in gene therapy.
Gene therapy vectors are capsules containing modified genome that carry a therapeutic gene in place of the viral genome.
In a process called transduction, infection introduces functional genetic information expressed from the recombinant vectors into a target cell.
In a successful gene therapy experiment the appropriate amount of a therapeutic gene ought to be delivered to the target cell.
The delivery of the therapeutic gene must be free of any toxicity along the way or damage, which tends to occur often.
An inherent set of properties that affect the virus’ suitability for a specific gene therapy application, are the characteristics of viral vector systems.
Every disorder requires different amount of genes and different systems of gene therapy, for example some genetic disorders require long term gene expression from a relatively small group of cell, whereas other diseases may require a high level of gene expression.
When doctors are try to interfere with viral infectious process, or inhibit the growth of a cancer cells, by the introduction of inactivated tumor suppressor genes, the cells may require the transfer of a large amount of genes to cover all the infected cells.
It seems to be a straightforward therapy when we use viral vectors, but gene therapy using viral vectors, still have many problems and risks.
Viruses are still not under total human control, because viruses may infect healthy cells as well as unhealthy ones, when the virus is inside the body.
The virus may also insert the new gene in the wrong location in the DNA, and this can lead to many complications for the patient, such as the development of cancer cells due to harmful mutations of the DNA.
Viruses may also infect a patient’s reproductive cells, when viruses are used to deliver DNA to cells inside the patient’s body.
Children could be affected by the changes, which may be passed on to the children, if the patient decides to have kids after the treatment.
Transferred genes could also be over expressed, which may result in a large amount of the missing protein being produce; this could lead to complications, because the large amount of the missing protein could turn out to be harmful to the patient.
The problems that exist today with vectors are being fixed by research doctors, because this basic mode of gene introduction into a patient’s cell shows many promises.
Before any clinical trials are conducted in humans, doctor are using animal testing and other precautions to identify and avoid risks that may put the patient at risk of losing their lives.
Nothing in life is without problems, and so it is the delivery of foreign DNA using viral vectors, but with better understanding of the human body and the life cycle of viruses, science doctors are working toward safer, more effective viral-based treatment that won’t put patient’s lives on the line.
The refinement of viral-mediated gene delivery protocols continues, providing a convenient, flexible and reproducible system for in vitro transfection studies.
Infectious virions are very efficient at transferring genetic information, based on the virus life cycle;
Type of Gene Therapy
In science-related news, gene therapy has become an important topic of discussion, because there are many promises that the introduction of health genes into ill cells has the ability to cure or prevent the progression of a disease, as normal functional copy of a gene is introduced into a cell, in which the gene is defective, with gene therapy.
Gene therapy is hoping to be able to eventually target the correction of genetic defects, eliminate cancerous cell, prevent cardiovascular diseases, block neurological disorders, and even eliminate infectious pathogens.
The two main type of gene therapy are somatic cell gene therapy and reproductive or germ-line gene therapy.
Somatic cells are every cell type in the mammalian body that does not form the sperm and the ova; internal organs, skins, bones, blood and connective tissue are all made up of somatic cells.
The insertion of new genetic material into the body’s cells and tissues other than the reproductive one, refers to as Somatic gene therapy; because science doctors want to make sure that the new cells introduced into a patient’s body are not passed on to the next generation if the patient decides to have kids after the therapy.
Effective means of DNA delivery systems changes the genetic coding of specific cells of a human body; the genetic alteration can be accomplished by using two methods In-Vivo or Ex-Vivo therapies.
Severe combined immune deficiency, muscular dystrophy, hemophilia and many other genetic disorders and acquired diseases are being cured, when medical professional use this type of therapy.
Germ Line Gene Therapy refers to the introduction of new genes into the reproductive cells and tissues of an organism.
Health genes are delivered to egg cells or sperm cells, to repair damaged reproductive cells in a patient’s body; which prevent defective genes from being transferred to the next generation.
The purpose of this type of gene therapy is to alter the genetic code of an organism and pass the good genes to the next generation.
Germ line gene therapy reduces the risk of defective genes being transferred to the next generation of the organism, promoting a more healthy population.
Science doctors may use germ line gene therapy by altering the genetic makeup of reproductive tissue before they are fertilized or they can alter genetic makeup of a blastomer at its early stages.
The purpose of germ line gene therapy is to target the genetic code of an organism and then change that genetic code, and then the new genes can be transferred to future generations.
Ethical and Social Consideration
Gene therapy would not be without controversy, it is as if we are playing God, but isn’t that what God himself would want of us.
For us to be the captain of this ship until he gives us another purpose in life, I mean if we were not meant to know and understand DNA to the point where we can manipulate it, what is the purpose in learning the creation and about our creator.
The problem with gene therapy though is the fact that there are many risks associated with it, which is why many scientists have developed a proposed experiment that incorporates strict guidelines, and gained approval from the FDA.
Gene therapy should not have more risks and problems than the potential benefits it can provide to anyone who uses it.
Even with the approval of the FDA many organizations continues to pay close attentions to clinical trials performed under gene therapy; because researchers are now required to report any harmful side effects.
Gene therapy also poses ethical concerns that people should take into consideration, such ethical considerations are: deciding what is normal and what a disability is; deciding whether disabilities are diseases and whether they should be cured; deciding whether searching for a cure demeans the live of people who have disabilities; deciding whether somatic gene therapy is more or less ethical than germ line gene therapy.
Treatments and experiment done in gene therapy should be tested extensively, many people can share that opinion.
Treatments that have failed on patients are usually replaced by gene therapy treatments, these patients may not have any other choice and need to move fast to safe their lives.
In the 1960s recombinant DNA research began and so did the public rage and public debate over using gene technology to treat human beings.
God has been the only being to successfully put in place a system of life that works and makes sense of you allow it to make sense; which is a reason why many people think that the human race should not be playing with the DNA.
Many people fear the worst results; if DNA technology was to fall in the hands of people, who meant nothing but harm to the human race.
However, gene therapy is a key to life, the coding of our existence here in life, I mean imagine if we had the knowledge to be able to manipulate DNA to rediscover a new type of body, an eternal body.
If we could make our body immune to any old and new disease we would and could live in that world.
But this is possible only with the guidance of sound and proper research, to be able to distinguish the right way and the wrong way of performing gene therapy on a person.
Many in the scientific community believe that this is a unique ground breaking advancement of this new science and technology; this is the most exiting application of science DNA, undertaken so far, because of the potential that gene therapy has to offer.
Some believe that injecting some body with DNA will solve all the genetic disease we have in the world.
I personally don't think so.
We should not be injecting people with DNA, even if the treatments are successful, because we don't yet its long term effects; but we are investigating the long term effects on mammals.
We should only manipulate the DNA that is already in the organisms body, change its chemical components, because the actual letter of the coding of the gene of an organism is unique to that organism.
But some of us believe that genes may ultimately be used as medicine and given as simple intravenous injection of gene transfer vehicle that will seek the target cells, for stable, site specific chromosomal integration and subsequent gene expression.
In other word, gene therapy is the way to go, by introducing new DNA in an organism's systems.
In general gene therapy is essentially good to life, and the advancement of humans as a race.
Our bodies are made up of the DNA which codes for the normal functionality of our bodies, and the knowledge of DNA is opening a lot of new worlds in our imaginations.
We may some day be able to fly and jump high, or heal quick and even live for ever with modified DNA.
Not so much with new gene but only the modification of our own genes; because it makes more sense that we would want to work with what we already have and try to fix that one.
Each organs of our systems form part of the body we carry around, we are actually put together in a very unique way, and we don't take care of our bodies and the organs we were given, we shouldn't be replacing them with new ones.
But we do it anyways, I do believe in gene therapy, but I don't believe in the replacement of an organism genetic code with new ones.
Time will tell though, we shall see how it works for every one to many humans turning into something we weren't meant to be in the first place, something that does the human race no good.
Or we may also want to turn into the super human that we see our selves be, jumping or flying to places, and living for ever, with out the need of becoming old, or losing lives.