Gene Therapy Research Papers

Gene therapy is a very controversial topic that has been discussed in the last five years and is being heavily studied to help cure cancer. Gene therapy is a technique aimed at treating genetic disorders by introducing the correct form of the defective gene into the patient’s genome (Dunlop et al., 2010).

DNA has about 12 million single nucleotide polymorphisms and thousands of copy number variants, most of which are not harmful. Sometimes these mutations in genes can cause genetic disorders that end up altering or inhibiting protein function. Gene Therapy focuses on correcting the mutated genes usually by inserting a normal gene into the genome. Researchers treat these diseases this way by repairing dysfunctional gene or by providing copies of the missing gene. They isolate the normal DNA and package it into a vector. Other ways include replacing the abnormal gene with a normal one, repair the mutated gene, or altering the regulation of a certain gene. As of right now, reparative results have been marginal, there is potential of gene therapy treating single-gene disorders.

     It is inevitable that eventually each of us will grow old and begin to face more and more health problems as our age rises. Elderly people are challenged by many illnesses and diseases that unfortunately, are incurable. One disease that becomes more common as people age is Alzheimer’s disease. Alzheimer’s a common cause and a form of dementia and can severely damage a patient’s cognitive functions and can ultimately cause death. Living with Alzheimer’s disease can be saddening for both the sufferer and the family. Family and friends will find it very hard to cope when a loved one begins slipping away and losing memory of who they are.

What is Alzheimer’s? Many believe they know exactly what it is and how it affects us, but in reality, many don’t realize how deadly it truly is. Alzheimer’s disease is said to be the most common form of dementia (Brandt). It is a disease that slowly kills thinking skills as well as memory and the ability to function properly. The deterioration and changes in the brain, however, can begin anywhere from ten to fifteen years before the signs of memory loss appear (CDC). Changes begin as, abnormal deposits of proteins form amyloid

There are more than 5 million people in the U.S. with Alzheimer’s and it causes 500,000 deaths a year in the U.S. alone. This disease is growing quickly, by the year 2050 it is expected that there will be 16 million people diagnosed with it in the U.S. and 100 million worldwide. The goal is to overcome this disease by the year 2025, which is possible, but very challenging. A neurologist at Harvard Medical School, by the name of Rudolph Tanzi said, “We will be able to identify those most at risk based on their genetics, do imaging tests to determine the onset and then institute therapies that nip it in the bud”(Marsa). This is great news because it will help the doctors get a jumpstart on those who are at risk of getting the disease. Memory loss is the main issue of Alzheimer’s which is caused by tangles in the brain. Without the tangles there is no disease, the tangles are what cause it. Alzheimer’s also causes shrinkage to the brain. Moving ahead with a cure, researcher need to figure out how the protective mutant gene works, making a treatment to copy what it does

Alzheimer's is a progressive disease that destroys memory and other important mental functions. As time goes on the symptoms get worse and unfortunately there are no cures available for Alzheimer’s. However, there are ways to “control” it.

In 1906, Alzheimer’s disease entered the scientific world. Till this day, it is one of the most studied neurodegenerative diseases. Researchers have come a long way with scientific outcomes on the disease, but unfortunately there is no official cure, or a concise reason on how this disease is generated. The disease has been recognized to being genetic and affecting people in their later years, roughly around their sixtieth year. Alzheimer’s disease affects the person’s memory, language, judgment and even their daily tasks. While the disease continues to dramatically progress, it begins to affect all regions of the brain, causing the person to lose almost all of their functions. When the person has reached their final stage, they are no longer able to recognize themselves or their surroundings and would need full time dependent care. According to the Alzheimer’s Association (alz.org, 2016), the person may have up to eight years max to live after diagnosis.

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).

Gene therapy is a rapidly developing technology first used in 1990.1 It involves the use of modified DNA inserted into an organism with a missing or mutated sequence. There are two methods of delivering this correction into the patient’s cells. In the viral method, a virus containing the vector with the corrected form of the gene, infects the cell naturally. Adenoviruses, adeno-associated viruses, lentiviruses, and retroviruses can all be used. They each utilize a different method of delivery and form of DNA. Adeno-associated viruses and lentiviruses are the easiest forms to construct.2 The corrected DNA faces many obstacles to gene expression and ultimately protein production. The viruses must target and enter the correct cell, avoid

In short, gene therapy is a method in which DNA Is used to fix a genetic problem. These problems can include missing or genes that don’t work. The right genes are delivered to the cell via harmless viruses for their provirus properties. The viruses then insert the good copy of the gene into the patients DNA where the cells machinery reads the information like a manual and makes RNA proteins so when it divides, ideally, the problem will be fixed. There are two types f gene therapy: Germline (targets egg/sperm cells; stops disease in its tracks) and somatic (targets somatic cells; after the disease is active).The methods for gene therapy that are being tested include:

One of the biggest contributors to the development of the gene therapy approach within medicine was the completion of the Human Genome Project that gave scientists and doctors critical information about the nature of human genetics. It thus became possible to gain a better understanding the connection between certain genes and the development of diseases and disorders linked to them. However, despite the progress that has been made since the completion of the project, there remains a lot still unknown about the human genome and its intricacies. The next generation of medical treatments that will explore this genetic realm will be sophisticated to a level never seen before. The promise and potential that gene therapy has for

Gene therapy, the experimental strategy that requires the use of genes to prevent and cure diseases is a fairly new technique, yet it is one which has shown steady advancements. The journal article “Gene therapy clinical trials worldwide to 2012—An update” sets out to present analysis and summaries of clinical trials that have been performed worldwide. Authors Samantha L. Ginn, Ian E. Alexander, Michael Edelstein, Mohammad R. Abedi and Joanne Wixon dive into some of the 1843 gene therapy trials performed in 31 countries. Analyzing geographical distribution of trials, searching for indications of mishaps in trials, speaking on diseases which have been tested in trials and a general introduction to gene therapy are some of the points within the article.

Researchers have been trying to find new ways to cure disease for centuries. Gene Therapy

Gene therapy could be an approach to alter a genetic issue at its source. By including an amended duplicate of a damaged gene, gene therapy guarantees to help ailing tissues and organs work legitimately. This methodology is not quite the same as conventional medication based methodologies, which may treat side effects yet not the fundamental genetic problems. Most generally, gene

Gene therapy, in a nutshell, is the use of genetic information from genes in the treatment and prevention of diseases to modify the phenotype of cells. According to Maria P. Limberis of the University of Pennsylvania, “prior to the publication of the DNA structure by Dr. James D. Watson and Dr. Francis Crick, Dr Clyde E. Keeler predicted in 1947 that gene therapy would be ‘a therapeutic technique’, ‘achieving permanent correction of hereditary diseases’. Delivery and expression of a therapeutic gene in an affected tissue or organ holds great promise for the treatment of various life-threatening acquired and inherited diseases that affect humans”[21].