Gene Therapy And The Treatment Of Alzheimer Disease

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).

Alzheimer's disease is one of the most common causes of dementia. The term 'dementia' describes a set of symptoms, which can include memory loss, changes in mood and problems with communication and reasoning. These symptoms occur when certain diseases and conditions, including Alzheimer’s disease, damage the brain. Alzheimer's disease could be described as a physical disease affecting the brain. During the course of the disease, protein 'plaques' and 'tangles' develop

Name: Ghida Krisht Informative speech outline 1. Topic: Gene Therapy 2. General purpose : to inform 3. Specific purpose: throughout this speech I aim to shed light on the effective technique of gene therapy.

Alzheimer’s disease (AD) is a progressive and fatal form of dementia, frequently seen in the elderly altering their cognition, thought process and behavior. AD is reported in about half of patients that have a dementia diagnosis; one study states that about 10.3% of the population over 65 years is affected by dementia with an increase to almost 50% over the age of 8 (Beattie, 2002). Alzheimer’s disease is not a normal part of the aging process in humans, but rather found in a group of diseases that affect the brain leading to a decline in mental and physical control. AD when diagnosed has a very slow and gradual course, initially affecting the individual’s short term memory (Beattie, 2002). Alzheimer’s disease is the 6th leading cause of death, affecting more than five million people in the United States and is also one of the most common forms of dementia. Dementia can be defined as a disorder of progressive cognitive impairment severe enough to affect daily functions of an individual’s life (Fillit, et al., 2002).

Alzheimer 's disease (AD) is a progressive degenerative disease of the brain from which there is no recovery. There are three brain abnormalities that are the hallmarks of the Alzheimer’s disease is initially caused by plaques buildup in the brain’s neurons as illustrated in figure 1. The support structure that allows the flow of the nutrients through the neurons gets damaged and ultimately there is loss of connection among the neurons and they die off (National Institute of Health, 2015). This causes the brain tissue to shrinks, which is called atrophies. All this ultimately lead the victim of this disease to face difficulties in governing emotions, recognize errors and patterns, coordinate movement, and remember. Ultimately, a person with AD loses all memory and mental functioning.

Alzheimer's Disease is one of the societal issues that Scientists will be able to address. Most Alzheimer's patients have gotten the disease from their parents, meaning, scientists can study the genome of the patients to understand why it is genetic. Alzheimer's is difficult to understand because someone can get the gene even when no one else in their family has it. Studying the genomes of these patients can also give light on why there is Early-onset Alzheimer's and Late-onset Alzheimer's. Through this study and research, scientists can get a better understanding on how to slow down or even cure the disease.

Luckily, there are biotechnology companies trying to solve the growing problems that Alzheimer’s disease is presenting to the public. Despite repeated failures in clinical trials, leading pharmaceutical companies continue to invest time and capital in finding Alzheimer’s disease treatments. 99 percent of therapies in the past decade have come up short. Scientists are bitterly divided on the issue of targeting amyloid to effect the progression of

Alzheimer 's is a slow and debilitating disease that generates multiple problems with cognitive skills including; memory, thinking and behavior. (MedlinePlus, 2015) It is especially painful for friends and family members who see their loved ones progressively lose their memory and ability to function as they normally have. Symptoms typically develop gradually and progressively get worse over time, becoming severe enough to put the person afflicted with the disease unable to complete daily tasks and placing themselves at risk. (MedlinePlus, 2015) Alzheimer’s is the most common diagnosed condition of dementia. (MedlinePlus, 2015) Sixty to eighty percent of dementia cases are designated as Alzheimer’s. (MedlinePlus, 2015) Although the greatest known risk factor for Alzheimer’s is increasing age, it is not considered a normal part of aging. The majority of those diagnosed with Alzheimer 's are approximately sixty-five and older. However, Alzheimer’s is not just a disease affected by the older age populations. About 5 percent of people with Alzheimer’s experience early symptoms and the onset Alzheimer 's and most often appears in their forties or fifties. As stated above, Dementia or Alzheimer’s symptoms gradually worsen over time. In the early stages, it is common to experience mild memory loss. Eventually, the ability to carry on a conversation is lost. The sixth leading cause of death in the United States is Alzheimer’s. After Alzheimer’s symptoms become publicly

Alzheimer's Disease is a condition that affects 50% of the population over the age of eighty five, which equals four million Americans each year. It is becoming an important and high-profile issue in today's society for everyone. There are rapid advancements being made in the fight against this disease now more than ever, and the purpose of this essay is to educate the public on the background as well as the new discoveries. There are many new drugs that are being tested and studied every day which slow down, and may even halt the progress of the disease.

Ultimately, the path to effective new treatments is through clinical trials (“Medication”). “Future Alzheimer's treatments currently work by temporarily improving symptoms of memory loss and problems with thinking and reasoning” (Mayo Clinic Staff). “Future Alzheimer's treatments are focused on combinations of medications like those used for many cancers” (Mayo Clinic Staff). An ongoing clinical trial conducted by the Dominantly Inherited Alzheimer Network (DIAN), is testing whether antibodies can reduce the growth of beta-amyloid plaque in the brains of people with certain genetic mutations and thereby reduce, delay or prevent symptoms

Gene therapy is a relatively new area of medicine that attempts to apply recent advances in molecular biology, genetics and biotechnology to the treatment of human diseases. Gene therapy uses a set of approaches to the treatment of human disease based on the transfer of genetic material (DNA) into an individual. Gene delivery can be achieved either by direct administration of gene-containing viruses or DNA to blood or tissues, or indirectly through the introduction of cells manipulated in the laboratory to harbor foreign DNA. As a sophisticated extension of conventional medical therapy, gene therapy attempts to treat disease in an individual patient by the administration of DNA rather than a drug. (1)

There is only one viable technique in gene therapy to humans – adding a gene to replace improperly functioning gene through a vector. Its basic mechanics is administering the DNA to the patient. Introduce the DNA to the diseased cell by a genetically engineered virus and express a protein. This can be done by injecting it to the bloodstream. The therapeutic genes are hoped to genetically alter the cells for normal functioning.

As the numbers of people suffering from CNS diseases are on the rise, treatment option still remains limited for many CNS diseases. Therefore, significant unmet medical need for these diseases allows AAV Gene therapy to transform the lives of many patients. The most significant aspect of AAV gene therapy is that, it is targeted therapy which could be an effective alternative medication. Moreover, gene therapy will have a durable and meaningful benefits to the patient.

1.Preface : Gene therapy, looks all previous fields of cruel edge scientific report, and was the subject of major ethical debate. Gene therapy has generated could be argued further discussion of other medical discoveries in its infancy because of its ability to influence the human genome. There are two types of gene therapy is currently under discussion, gene therapy and somatic cell gene therapy from germ cells. 2. Point of view for gene therapy research:

The first, involving gene replacement, attempts to correct a specific genetic defect linked to a major component of the disease by expressing unmutated gene. In reality, the number of patient whose neurodegenerative disease is linked to a single genetic mutation is quite small, so the impact of this application of gene therapy to neurology is limited. Another goal of gene therapy is to transfer therapeutic proteins to a targeted area of the central nervous system (CNS), not