Gene Therapy Biology Assignment FINAL COPY
By Hunter Alker 9S
For countless generations, genetic diseases have been ruining people’s lives with these abnormal conditions being able to severely mutilate, disable or kill them. Genetic illnesses are most commonly due to mutations in the DNA sequence that occurs during replication or due to environmental factors such as radiation. [10] Though an affected patient might not have to live with their illness for their whole life as the human genome has the ability to be changed [10], therefore, allowing an opportunity for treatment. Research and advancements in technology have allowed numerous different methods of treatment for affected patients to be discovered which have dramatically changed our
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Having a cure for genetic diseases would be a significant progress in the field of medicine. [3]
In the years around 1960-1970, the idea of gene therapy had been arising as a possible solution to curing some genetic disorders, although reliable data about the safety and consequences of the process were still being debated. In 1890, the NIH Clinical Centre housed the first patient to be treated with gene therapy, a four-year-old girl who suffered from a disorder called adenosine deaminase (ADA). The girl’s white blood cells were taken away and then infused with the correct gene. [2] This treatment was undertaken repeatedly for two years and when the gene therapy had ceased the patient’s health had significantly increased and the presence of the ADA enzyme had decreased dramatically. Around the same time, a nine-year-old girl also with ADA received the same treatment with similar results. [4] After these early successful treatments using gene therapy, scientists then used this method of genetic engineering to remedy babies with this disease before expanding to the treatment of other diseases.
Originally, our understanding of genetics was commonly utilised in the form of genetic testing which allowed scientists to identify and diagnosis genetic disorders and diseases by observing chromosomes, genes and proteins. This limited out knowledge to
When used in healthcare, genetic testing can show the genetic cause of a disease, confirm a suspected diagnosis of an illness, predict future illnesses, and predict responses to
Although the treatment is costly and only available in clinical trials, gene therapy has treated some of the most known diseases, like Parkinson’s disease. People who are for gene therapy believe that it will change our world by getting rid of sickness. They think of a world without cancer and Parkinson’s disease. However, the people against gene therapy see a world with technology overtaking civilization. In their eyes, they think that gene therapy is wrong because of the side effects and the concept behind it. In addition, when thinking about gene therapy, the opposers conjecture that changing the genetic makeup of someone affected with genetic illness is wrong. I, on the other hand, believe that changing the genetic makeup of someone that is ill and suffering is exceptional because it is to their advantage. All in all, gene therapy is a heavily debated topic, but I believe that gene therapy can change our world in a positive
Gene therapy is another approach to eradicating genetic diseases. “Gene therapy was considered for a long time appropriate only to treat monogenic diseases by means of a therapeutic gene transfer for replacement of a defective homologous gene.” (NPR 1) It is a very efficient way to prevent genetic diseases such as Crohn's. Modern day technology and scientists are curing many genetic diseases around the world using many forms or genetic
This is one of the major factors that lead to the absence in the gaining of therapeutic science. Although the lack in progress cannot be the only factor blasted for; some scientific trials have confirmed both anxious and fearful, advancing tighter on gene therapy restrictions. According to Hogarth, this hotly debatable topic of whether it is dangerous or not “playing God” has caused many religious, governmental, scientific and public figures to signify their own concerns in fierce debates (Hogarth para 9). Since the beginning of gene therapy, there has always been a constant battle over the guidance of its future of whether gene therapy will transform into a therapeutic science or lead to unpleasant consequences as the eugenics did in the 20th century. The ethics of gene therapy have been established and based on, not only beliefs, but also the disconnection and advantages achieved in the trials, which has lead to the breakthrough of the future of gene therapy. The downfall and advancement of gene therapy are the hereditary products of research that have had a major influential impact on its ethical features. When gene therapy was a novice, it gained an immense amount of popularity when scientific research evolved towards identifying genes that induce certain diseases (Hunt, 2006).
For future findings, figuring out a way to protect the chromosomes from mutation will prevent this disease from harming anyone and could lead gates to curing other diseases involving mutation.
Fifty years after the idea of gene therapy was first proposed, gene therapy has become a possible treatment for a couple different diseases. Before this treatment was approved, some serious unfavorable effects were found in clinical trials. However, these effects fueled more basic research in order to improve, in efficiency and safety. Gene therapy has been used for patients with blindness, neuromuscular disease, hemophilia, immunodeficiencies, and cancer.
In 1993 a pamphlet by National Heart, Lung, and Blood Institute was released heralding Gene Therapy. Although gene testing had been around and used for various procedures and breakthroughs, gene therapy had the potential to change the face of research, as we knew it. Medical scientists had finally found a way to
Gene therapy is an experimental form of treatment, where normal genes are placed into missing or defective ones in order to correct or alter genetic disorders. In other terms, it is the therapeutic delivery of nucleic acid polymers into a patient 's cells as a drug to treat disease (Gennady, 2015). After many attempts at modifying human DNA by nuclear gene transfer as well as insertion of human DNA in the nuclear genome, the first successful gene therapy was in 1990 where it was used to cure four-year-old Ashanti DaSilva who had Severe Combined Immune Deficiency (IDF 2013 National Conference). Since then gene therapy has been used to cure and treat serious diseases such as Chronic Granulomatus Disorder, Hemophilia, and a wide variety of Cancers, and other genetic and acquired diseases (American Society of GENE & CELL THERAPHY, online).
Scientists and doctors alike have long been looking for cures to diseases like cancer. For years now, there have been countless fundraisers to cure this and cure that. And yet, it seems as if there's never any progress. There may be a brief headline in the news about a new discovery, but they always seem to never go anywhere. Maybe the problem is that they've been looking in the wrong places. Maybe the cure doesn't lie on a medicinal level, but instead the genetic level, in genetic engineering and gene therapy. Direct manipulation of an organism's genome can be used in gene therapy as a means to alter genes, causing both positive and negatives effects as well as discussions about the ethical boundaries of modern
“In 2013, more than 43,000 adults and 5,000 children and teens will be diagnosed with [leukemia] in the United States” (“What you”). Leukemia is a cancer that affects an individual’s blood and bone marrow by making leukemia cells. Leukemia has many treatment options, but these treatment options are not guaranteed to completely cure cancer (“What you”). Doctors have been working diligently to find a cure for leukemia, and cancer in general. Some of these treatment options have included experimentation with gene therapy. Gene therapy is a treatment that goes to the source of the disease and helps correct the defective genes rather than treating the symptoms like traditional drug-based approaches (“What is”). Although gene therapy
Due to the fact that most incurable diseases/disorders are caused by the genes of a person, DNA modification can be used to create the best set of genes necessary to fend off any type of disease. Eventually if this type of genetic engineering is used on most of the world population, diseases such as cancer, Ebola, AIDS, and polio would completely disappear. Disorders like schizophrenia, diabetes, and asthma could also be wiped out of the gene pool altogether. Thus, the health of the future generations to come would be exceedingly enhanced.
Within this field, somatic gene therapy—the replacement of a faulty gene with a functioning one—is particularly fascinating. One of its interesting aspects is the ability to be customized to the individual. For example, not all patients suffering from a given cancer will have the same mutations. After sequencing a patient’s genome to detect the carcinogenic mutations, doctors can use gene therapy to target and treat the illness at its source. However, at this stage of the technology, gene therapy provides only short-term remission. Studies on blindness showed that the initial success of gene therapy faded over time—an unsurprising outcome since the cells of most tissues will die and be replaced by new, untreated ones. With this area of research still in its infancy, much remains to be done to improve the long-term effectiveness of gene therapy and make it a viable treatment. A potential solution would be to use gene therapy on stem cells as well as on specialized cells. The daughters of the treated stem cells can then differentiate, still carrying the therapeutic gene, and replace the original treated cells as they senesce. For so long, genetic disorders have been seen as something permanent, something scientists could not change. Changing that reality would be immensely
Here in the year 2017, we are very fortunate to be living at a time of medical advancement. Ailments and illnesses once thought to be death sentences centuries or even decades ago can be cured by a simple pill or vaccine. With that said, scientists and doctors still do not have all the answers. While we are much less likely to die of common diseases, there are some that are not so easily combatted. Some afflictions are difficult or near-impossible to treat, and this is especially arduous for those who are born with them. People suffering from genetic diseases
Researchers have been trying to find new ways to cure disease for centuries. Gene Therapy
Every living thing is the product of the genes that were passed down from ancestors. Genes make up everything we are. One gets their traits from their parents. Most people live full lives with relatively good health. However, some people inherit mutated genes or faulty genes. This could lead to genetic disorders that could be life threatening. Even today, many genetic disorders still remain incurable, leaving many people without hope. Genetic therapy could be their answer. It is through this research that the cure for genetic disorders can be found. Though some people believe it is unethical or immoral to alter genes, current therapeutics have not been able to save the lives of the patients with these diseases. Genetic therapy