Gene Therapy Essay

Gene Therapy Essay
Gene Therapy
I. Introduction
With the human genome project now completed, identifying our DNA, the next step forward is being taken to analyze this information and apply it in
a helpful context. As we discover which genes affect and trigger the different traits humans possess, new questions result pertaining to potential
problems in our DNA as well as genetic enhancement opportunities. In theory, once a problem is pinpointed within a person's DNA, there exists a
possibility for correcting this defect. Gene therapy is a technique used for correcting defective genes responsible for disease development. This
relatively new idea sparks much controversy when societal implications are examined. Bill Joy, chief scientist and...show more content...
The former concentrates on treating the individual, while the latter aims to eliminate "bad" genes from the individual as well as their offspring.
Somatic gene therapy up to this point has been used to treat rare, deadly, genetic diseases by the insertion of specific genes into human cells other
than the cells directly used in reproduction. One problem is the method of insertion. The methods that transfer the new DNA to a defective cell are
referred to as vectors. The most commonly used vectors are viruses, more specifically, adenoviruses or retroviruses ( Adams , 2004). Because of the
viruses' natural ability to get by the body's immune system and introduce DNA into cells without being detected right away, researchers find them to
be a particularly useful tool. First the virus must be stripped of its harmful properties and then forced to take up the corrective DNA. The remaining
goal is successful delivery to many cells in order for replication as well as insertion into the correct place in the chromosome.
Delivery can be categorized as ex vivo or in vivo. Ex vivo, the first delivery method used, removes cells from the body in order to modify and correct
the DNA. Through the aid of a vector and after cell modification, the cells are transplanted back into the patient where they will replicate. Blood or
liver cells are good candidates for this method ("Gene Therapy", 2004). In vivo
Get more content on HelpWriting.net

A Study On Gene Therapy Essay
Gene Therapy
I first learned about gene therapy while watching an episode of Grey's Anatomy. Over multiple episodes, Dr. Bailey is trying to cure a child's lack of
immunity with the use of gene therapy, and more specifically with the use of HIV as a gene transfer vector. To make the virus usable, Dr. Bailey
disables the virus and uses it for a traveling mechanism. The parents were afraid that disabling the virus would fail, causing their child to be infected
with the HIV virus, so they pulled their consent for the operation. Dr. Bailey decided to do the operation anyway because she's awesome and she
knew she could save this child's life. She risked her career and good standing in the medical field, but she was ultimately successful in curing the child.
This sparked my interest in gene therapy and all of the possibilities and progress. (Barnett).
Gene therapy is the transfer of nucleic acids to a person's cell to correct a defective gene or cure a disease. The treatment involves actually replacing
faulty genes. In the case of hereditary and other lifetime illnesses, it offers a chance of curing a patient permanently of their ailment. After the
corrective genes have been replaced in the gene transfer vehicle, the vehicle is injected into the patient, with ex vivo or in vivo injections (Kumar et
al). Gene therapy has been around for over two decades. It has had setbacks, but thanks to the advancement of technology and medical understanding,
has become more effective in

Gene Therapy Essay
Scientists believe in Gene Therapy Can Treat Cancers? Sarah Dziatko
For many years treatment for cancer has been radiation, chemotherapy, and surgery, but through clinical trials immunotherapy has been tested on
various patients that the previously listed treatments did not work for. Immunotherapy is a type of therapy in which the immune system is altered and
strengthen to become more rigorous and able to attack cancerous cells. This new discovery is extremely important because it is like a fifth pillar in
cancer treatments providing some type of result for doubtful patients.
Crispr is a new tool developed to edit genomes. This tool is significantly more precise, efficient, and flexible than technology previously used. Because
of this...show more content...
There are several types of ACT, but as of August of 2017 the only therapy that has been approved by the Food and Drug Administration (FDA) is
T–cell therapy1. Before this therapy was approved T–cell therapy was only available through small clinical trial for people with advanced blood
cancers. These trials had remarkable responses for adults and children whose other treatments failed. This got the attention of many researches who
worked to get this therapy approved. Now children with acute lymphoblastic leukemia (ALL) and adults with advanced lymphomas have the option of
gene therapy to stop the growth and spread of their cancerous cells1.
Gene therapy is an amazing advance in science and medicine, but it does have its drawbacks. Since a gene cannot be directly inserted into a cell a
vector is used as a carrier for this gene. Usually a virus is used as a vector because it can more easily recognize certain cells. The most common issues
are unwanted immune system reactions, vectors targeting the wrong cells, infection caused by the virus, and the possibility of creating a tumor if the
genes get inserted in the wrong spot in your DNA3.
Although these are major issues that could occur there are possible complications with even the simplest procedure. All gene therapy is overseen by the
Food and Drug Association (FDA) and the National Institutes to ensure that these therapies are

Essay on Gene Therapy
Gene Therapy
Parents can now pick a kid's sex and screen for genetic illness. Will they someday select brains and beauty too?
In the ever– advancing technological world, scientists discover new and efficient ways to advance society each and every single day. Imagine being
able to choose your child's body type, or personality, or IQ. It is not as farfetched as it sounds. It's a process called "Gene Therapy", and is being
perfected right now. This process rules out any unknown's in childbirth. It will not only allow us to determine the child's sex, but also his future.
In natural child conception, the mother provides the the two X chromosomes and the father provides the X and Y chromosomes. The balance of
genetic make up is...show more content...
Babies manufactured in test tubes would eliminate the need for women, and since most country's do indeed favor men over women, the world may
very well be run by males.
ETHICAL ISSUES
Who is to say it is right to deprive a child of it's natural existence?
The child, born and created through genetic manipulation, will still be human, but will live in a synthetically altered reality. A human, born or unborn,
has the right to have his/her own identity, naturally created by a mother or father, not a scientist. What right do we (science) have assuming that we
can step in and "takeover" mother nature and the natural process? If something is natural, that is the it is supposed to be, the way it has always been.
Complications thus far in the natural human life, consist of infant disease and death. It seems the humane thing to do to try to save these children from
this unpleasant fate, but that is reality. If we alter reality we are altering our world, and who we are.
Who Gets the Good Genes?
In Aldous Huxley's 1932 novel "Brave New World", childbirth is controlled by science and the government. The book relates the idea of reproduction
for the sake of social efficiency, to the natural way of birth. Creating babies from test tubes, and deciding one's existence on the basis of the betterment

of their society, is the controversial topic of

Gene Therapy
Gene therapy is an experimental technique that uses genes to treat or prevent disease. Currently, there are a lot of studies and experiments that allow
doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or direct surgery. Researchers are testing several approaches to
gene therapy, such as replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating, or "knocking out," a mutated gene
that is functioning improperly, introducing a new gene into the body to help fight a disease, and helping to regenerate precious cells. Although gene
therapy has a very active and promising approach for a number of disease, there are still several risks and side effects. Hence, the therapy...show more
content...
Light passes through the cornea, and is refracted and focused on the retina. The retina is a thin, delicate tissue located at the back of the eyes that
contains photoreceptor cells that detect light and convert it into electrical signals that are sent to the brain. The photoreceptor cell relies on a protein
encoded by gene RPE65 for the production of a type of vitamin A that allows light sensitive photoreceptor cells to function. The RPE65 protein is
produced in a thin layer of cells at the back of the eye named retinal pigment epithelium. About 10% of people with Leber congenital amaurosis
(LCA), an inherited disorder that causes vision loss starting in childhood, have an altered form of the gene RPE65. The researchers hoped to halt this
process by using a virus to shuttle a functional RPE65 gene into the retina. This strategy, in which genes are used to treat or prevent a disease is
known as gene therapy. Clinical trials of experimental gene therapy for LCA and other diseases that affect the retina has been progressing in the past
decade. In 2007, researchers at the University of Pennsylvania and the University of Florida pinpointed an area of intact photoreceptors in the retina
of 3 patients aged 22, 24, and 25. They then injected healthy copies of the RPE65 gene under the retina in this area. The phase one of clinical trial is
also supported by the NIH's National Eye Institute

Human Gene Therapy Essay
Human Gene Therapy
Image what it would be like if doctors could cure Huntington's disease, muscular dystrophy, or even hemophilia. Could this be possible? With gene
therapy this all could be reality in the near future. Gene therapy is a potential approach to the treatment of genetic disorders in humans. This is a
technique where the absent or faulty gene is replaced by a working gene, so the body can make the correct enzyme or protein and consequently
eliminate the root cause of the disease (BIO, 1990).
There are two types of gene therapy treatment: Somatic cell gene therapy and germline therapy. Somatic cell gene therapy involves obtaining blood cells
from a person with a genetic disease and then introducing a normal gene into the...show more content...
And also how do you make the delivery specific and safe? The first step in gene therapy is an accurate diagnosis of the genetic defect. This is done by
using a DNA probe. The DNA probe is specific to a complementary piece of DNA. This technique using DNA probes is more specific then other
conventional methods of diagnosing genetic defects in humans (BIO, 1990). After the correct diagnosis is made, healthy DNA can be inserted into
a virus, which has had infectious genes stripped out of it. The virus is then mixed with cells taken out of a patient and then injected back into that
patient. Viruses are used because they are like genes, but wrapped in a special protein coat. On the surface of this protein coat are specialized
proteins that bind to the surface of cells. Once these viruses are in the body they lock in place on specific cells. The cells then suck the viruses in or
the viruses force their way into the cells (PBS).
A retrovirus is a type of virus that is used in gene therapy. This is a virus that inserts its genetic code directly into the chromosomes of the host cell
(PBS). Other viruses are used for different kinds of genetic problems. One of these is the adenovirus which is used for cystic fibrosis patients (Coutts,
1998). To use a virus in gene therapy, the patient's immune system has to be weakened so it will not fight off the virus. If weakening the patient's
immune system is not

Gene Therapy
How can Gene Therapy be used to Treat or Prevent Disease?
Discussion
In the last two decades, gene therapy has improved from mental concepts to clinical trials and laboratory experiment. Gene therapy is a technique that
is currently being tested to see if genes can be used to treat or prevent disease. This method can successfully be able to replace a mutated gene with a
healthy copy, knock out a mutated gene that is not functioning and introduce a new type of gene in the body to help fight a disease a human being
may have. Gene therapy is a work in progress system that basically inserts a "normal" genome to replace the gene that is "abnormal" and causes
disease. Gene therapy have shown magnificent progression in animals. In mice, it has...show more content...
Women may not want to get pregnant because they are aware they have a genetical mutations related to mtDNA. Researchers wanted to see if they can
use gene therapy to prevent births with genetic disorders. They replaced the mtDNA in an egg from a healthy donor. The stem cells were not successful
for humans but did work better on monkeys. This technique was experimental and showed the potential gene therapy has.
Gene therapy testings are not always successful when experimented on humans. However, the technique has been effective on animals. Scientists used
gene therapy to change EphB2 levels in mice. The study showed that once levels were reduced they had memory problems like mice with Alzheimer's
(Roan, 2010). Increasing the level of enzymes in mice prevented memory loss. The use of gene therapy showed that amyloid proteins binds to EphB3
and causes the level to increase or decrease.
Conclusion and Future

Essay on Human Gene Therapy
Human Gene Therapy
Imagine this, you have just married your college sweetheart this past summer. You have now been happily married for over a year and you find out
that the two of you are going to be parents. So as the typical soon to be mom and dad so commonly do, the two of you start going to the doctor for
checkups to make sure the pregnancy is running smoothly. Early in the pregnancy you hear the worst, your baby has a fatal disease. The two of you are
terribly heart broken over the situation. You tell your doctor that abortion is out of the question because you do not believe in it. On the other hand the
doctor hits you with the question, "What about gene therapy?" Unfortunately you and your spouse look dumbfounded at each other...show more
content...
Gene therapy techniques consist of several key elements. An important element, actually one of the most important elements is the identification and
cloning of the gene or genes related to the disease, which is going to be maintained or treated by gene therapy. However, gene therapy should not be
confused with the concept of cloning. Cloning is creating another individual with essentially the same genetic makeup. The idea of cloning an entire
human is very different from gene therapy. Next the expression and efficiency of gene transfer during gene therapy must be emphasized. Scientists
involved with gene therapy research are currently trying to discover the best rates of gene transfer and tissue–specific gene expression. Gene therapy
research is still in the formative stages. Although a number of early human clinical trials have been initiated to test important concepts that have
emerged.
The genes we inherit from our parents virtually affect every human disease. The argument for gene therapy lies in the understanding of the genetic
basis of human disease. There are an amazing 100,000 individual genes in the human genome. At the present time there is an international movement
known as the Human Genome Project. The project will eventually provide an understanding of how each gene plays a role in human lives. The project

Gene Therapy Essay
Gene Therapy
Gene therapy is a powerful new technology that has the ability to change the way medicine is practiced in the future. The potential of gene therapy
offers great hope for cure and alleviation of suffering from genetic disorders that now plague numerous people. Within this past decade, much research
has been conducted to learn about the aspects of gene therapy, but there is still much to learn before it is an effective medical treatment. Despite
failures to prove any clinical efficacy, many experts ofgene therapy predict that the first clinical success will occur in the near future. Gene therapy is a
highly controversial topic that entails numerous ethical issues that need to be thoroughly analyzed before it is widely...show more content...
One type is called somatic gene therapy, which involves the manipulation of gene expression in cells that will be corrective to the patient but not
inherited to the next generation. This is the type of gene therapy that is currently being intensely studied in laboratories throughout the world. The other
form of gene therapy is called germline gene therapy, which involves the genetic modification of germ cells that will pass the change on to the next
generation. Little, if any, research is currently being conducted in germline intervention largely for technical and ethical reasons. However, many
advocates of this type predict this will be a realistic option of gene therapy in the future.
How does gene therapy work?
When research on gene therapy began, the basic challenge was to develop a technique for delivering genetic material to the cells of the patient.
Researchers first learned that gene delivery would not be effective unless the corrective genes were inserted into the nuclei of thousands or millions of
diseases cells (Licking 96). When researchers simply injected the genes into the specific tissue where they were needed, no treatment occurred because
the genes did reach the cells nuclei (Licking 96). Researchers learned that a therapeutic gene must be delivered by a gene–delivery system or "vector"
in order to be inserted into the target cells of the patient's body.
Many of the vectors

Gene Therapy Research Paper
A New Future: Gene Therapy
Imagine a world where a person could change his or her genetic structure and redirect the future course of evolution in their child and themselves.
Through gene therapy this is a very real possibility. In the future it could be just as easy to change your physical or mental health as it is to get flu shot
now. But the affects of gene therapy are long lasting and could affect your future offspring as well as your own health.
What is gene therapy?
First discovered in the middle of the 1970's researchers were able to isolate certain genes from DNA. During the 1980's the term gene therapy came
about and propelled research further.
The definition of gene therapy is a "technique where the genes causing a...show more content...
This is because any rearranging of genes happens in the somatic or non–reproductive cells (BIO, 1990).
The net type of gene therapy takes place in the reproductive cells or germline cells. The term germline therapy is used to describe the form of gene
therapy where the correct gene is inserted into the reproductive cells. Therefore when the treated individual reproduces his or her offspring will have
the correct gene instead of the defected one. This treatment affects both the parent and the offspring. It is also possible to insert a manipulated gene
into an early embryo so this new individual will have the correct gene sequence. This treatment only affects the embryo. One thing to remember is that
germline therapy not only affects the individual but their potential offspring as well (BIO, 1990). Thus germline therapy is passed on into the gene
pool of future generations.
The challenge in gene therapy is how to get the correct genetic material to the appropriate cells. According to Encyclopedia Britannica online 1998
there are three types of delivery systems– a viral vector, a chemical method or a physical method. The most common and accurate is a viral vector.
Technology has developed vectors, which encapsulate therapeutic genes, in order for delivery to defected cells.
Many vectors are actually viruses. Viruses are efficient in targeting cells and delivering information. According to PBS online 1998, viruses are
actually genes wrapped in a protein coat.

Gene Therapy Essay
Gene Therapy
The Human Genome Project began around 1986. The main goal of the project is to locate and sequence all genes found in human DNA. The
objectives for this ambitious effort are to learn more about heredity of disease and to discover the genes that would aid in gene therapy. Advances in
gene therapy strive to treat hereditary diseases and possibly eliminate disease from the genome.
The definition of gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases (Jaroff, 1996). Gene therapy
has two possibilities of disease treatment, somatic gene therapy and germline gene therapy. Somatic gene therapy involves the manipulation of gene
expression in cells that will be corrective to...show more content...
Possible vectors are retroviruses. Retroviruses are specialized virus containing RNA that has a knack for finding its way into a cell's genome and
making itself at home (Elmer–Dewitt, 1994). This vector is optimal because it can be genetically engineered to not infect the patient with the virus, but
it will carry the corrected gene in the RNA. When the retrovirus attacks affected cells, it will commence replication, replacing the affected gene with the
corrective gene. The healthy cells are then injected into the patient to continue replication in the body.
An example of this procedure was first demonstrated in 1990. The patient was a four–year–old girl. She had severe immune–deficiency disease caused
by a faulty gene that failed to order the production of a vital enzyme. (Jaroff, 1996) Some of the white blood she carried were extracted and
introduced to genetically engineered viruses containing healthy copies of the gene. The viruses performed as expected and attacked her white blood
cells. The cells were then injected back into her bloodstream. The newly corrected genes began producing the proper enzyme. The problem that arose
with this particular procedure was she was not cured, because the altered cells did not reproduce and eventually died. They young girl needed
treatment to be administered every one to two months, based on the lifespan of the cells containing the corrected gene. Treatment did make it possible
for this child to lead a relatively normal

Gene Therapy Essay

Recommended

Recommended

More Related Content

Recently uploaded

Recently uploaded (20)

Featured

Featured (20)

Gene Therapy Essay