In this study I will be observing the effectiveness of Gene therapy for Parkinson’s Disease. This disease has been affecting the lives of 60,000 Americans each year. With the hardship of going through surgery and drug medication, it’s time to think about having gene therapy as an alternative for patients. Its very important to know the foundation of the disease and how it is developed. There will be many cases and studies done to prove the effectiveness of gene therapy, how it helped other diseases and in the future Parkinson’s. The most successful experiment was with the AAV2 vector, that created promising results. A group of subjects 60 years or older were lucky enough to receive gene therapy treatment that relieved them of their symptoms.IntroductionThe focus of this study is to reveal the effectiveness of Gene Therapy. It is a very powerful experiment that we will be seen throughout our future. In gene therapy, there has been proof of success with humans and even more outstanding, animals as well. Scientist are figuring out how this experiment will work on certain diseases and creating better results. Gene therapy aiming to completely eliminate symptoms and not just have them in remission (Stoessl 2007) The purpose of Gene therapy is the addition of new cells in the body that will serve as a replacement for damaged cells. What doctors do with their patients is they inject them with a virus carrying that genetic cell. They will inject the virus directly to the part of the body with that cell deficiency. This is useful when certain cells need to be fixed and great for parkinson’s disease because only a certain part of the brain needs to be targeted. In gene therapy for Parkinson’s Disease a population of genes are genetically altered to relieve symptoms, or restore the genes normal function. There are many methods to target the cells such as nonviral methods. This method is used for the transfer of genes to the central nervous system or physical methods where you use a gene gun or electroporation. We also have viral vectors where genes can be removed from their genome. Vectors are now able to infect cells, but not replicate them. The adeno-associated virus, referred to as serotype 2 (AAV2) vector, has managed to be a safe vector when targeting the region of the brain when using gene therapy.Review of Literature Parkinson’s disease is one of the most common movement disorders affecting 60,000 Americans over the age of 60. The disorder is the result of insufficient dopamine producing cells in the substantia nigra. The current treatments are dopamine enhancing drugs, or brain surgery to improve symptoms. In this study we will be doing a before and after study on the effectiveness of gene therapy as an alternative to relieve Parkinson’s symptoms.The neurological disorder first introduced to as ” shaking Palsy” affects the region of the brain called the substantia nigra. The substantia nigra are group of neurons in the midbrain that are responsible for the production of dopamine.(Clarke 2007) . The dying neurons is what produces dopamine, the chemical that sends messages to the part of the brain that controls movement and coordination (Clarke 2007). Identified by researchers at the Florida Hospital at least 1 million people suffer from Parkinsons. There are about 60,000 cases a year. Furthermore, the average age for Parkinson’s is 60 and high percentage of the people are men.Parkinson’s has a variety of symptoms called primary, secondary and non-motor symptoms. Primary symptoms Resting Tremor is the most important because around 70% of people with Parkinson’s will have it (Clarke, 2007). Bradykinesia or ” slow movement” can give the appearance of abnormal stillness and decrease in facial expression. Furthermore, there is rigidity or stiffened flexibility of the limbs, neck and trunk (Clarke, 2007). Postural instability is another important symptom, which is the tendency to be unstable when standing up. Now, secondary symptoms can be an example of freezing, hesitant to step forward or unwanted acceleration. Non-motor symptoms Loss of smell Bladder problems rapid eye movement (REM) sleep disorder. There is also orthostatic hypotension where you have low blood pressure when standing up (Clarke, 2007).Like any other disease Parkinson’s has its stages of progression. Stage one is the initial phase where people experience mild symptoms. Stage two The symptoms are bilateral where they start to affect both limbs of the body (Clarke, 2007). Stage three symptoms are severe and includes the inability to walk straight or stand. Stage four you will experience severe symptoms like bradykinesia and rigidity. They are not able to complete day-to-day tasks. Lastly, is the final stage, they can no longer take care of themselves. They now require one-to-one nursing care.Now,the causes of the diseases will be examined. It is discovered there could be genetic and environmental factors that play a role in the development of the disease. Environmental Factors are toxic chemicals, viruses, bacteria, or heavy metals that can cause dopamine producing neurons in the substantia nigra to die. A good example would be, According to the European Parkinson’s Association, around the 1980’s drug addicts in California took heroin that had been contaminated by a hazardous synthetic drug called MPTP. The people who took the drug began to develop parkinson like symptoms almost immediately. Next are genetic factors contributing to the development of parkinson. It is very rare to pass on the disease from parent to child. If you have a parent with the disease the chances of developing it are as low as 9%. In recent years there have been a number of cases where parkinson’s disease has been considered genetic. A mutated gene has been passing on from one generation to another with people in the same family.The treatment for Parkinson’s disease has not been progressing, especially the medications. The medication Carbidopa or levodopa remains the most effective drugs. Levodopa is a more potent drug, but they combine it with carbidopa to reduce the symptom of nausea and vomiting that comes with levodopa (Mestre). There are also dopamine agonists that are injected and stimulate the brain into thinking it’s receiving the dopamine it needs(Mestre). . The surgical treatment is deep brain stimulation. They insert electrodes into a targeted region of the brain and monitor the brain cell activity.Gene therapy is the experimental technique that will allow doctors in the future to treat a disorder by inserting genes into the cells instead of surgery or drugs. How this technique works is by introducing genetic material to the abnormal gene to produce the beneficial protein that the mutated gene causes a shortage (Douglas, 2013). The gene is not directly inserted because that generally never works, so scientist use a carrier called a vector. A vector can be a non harmful virus that is injected and that virus will infect the cell and deliver that gene instead. The patients cell will be removed and exposed to that vector before actually performing it. As previously stated, the treatment for Parkinson’s disease has not been progressing, especially the medications. Effective medical management for the disease could be dated back to the 1960’s (Coune, 2012). Adults that struggle with Parkinson’s disease don’t have numerous alternatives when it comes to choosing what they want to reduce the symptoms of the diseases. When it comes to relieving treatments you don’t really have a choice, either you take medications with side effects on top of your or go through a riskful surgery that may not even have good results. People are taking their chances and have to settle with only having to options (Muramatsu 2010). This is where gene therapy comes in. It is proven to be effective for later stages in Parkinson’s disease and eventually will eliminate other treatments (Douglas 2013). The questions raised with the effectiveness of gene therapy for Parkinson’s disease is the vector that will be used and how to deliver that gene to the targeted area. The adeno-associated virus, serotype 2 (AAV2) vector managed to answer some of those questions (Douglas 2013). The virus is very neuron specific, but can cause a slight hormone response when the AAV has escaped the brain (Douglas, 2013). Since there is more experience with the AAV2 vector it is enhanced that it is completely safe .Clinical Investigations for parkinson’s using gene therapy have advanced with no confirmation that using the AAV2 vector isn’t safe. Research groups investigated four transgenes in conducted trials. They used gene therapy for enzyme replacement and fifth group used a neurotrophic transcription factor. The glutamic acid dehydroxylase (GAD) gene with AAV2 vector to brain stimulation, of PD patients in both Phase I and II trials prompted the discussion of proceeding Phase III trial conclusive.This was the gene therapy experiment approach that is promising to completely relieve parkinson, but even more success came when Professor Mazarakis entered Paris and witnessed monkeys lacking movement in their cages. What came to mind was the use of gene therapy. The surgeons could not be any more surprised with the results. The treatment had corrected the movement deficiency in the monkeys. After verifying that the animal experiment was safe to test on humans, scientists didn’t hesitate to try. They performed the same experiment on humans by injecting the region of the brain lacking those important dopamine producing neurons and were astonished by the results. The patients’ scores in the movement category improved by 30 percent, and were able to have a better quality of life. The first patients to have the surgery were followed up for four years, and the effect was sustained. The Positron Emission Tomography (PET) scans confirmed that dopamine is being produced in the brain where it wasn’t producing before.MethodologyTo find the answer of whether gene therapy will be an effective approach to completely eliminate the symptoms of parkinson’s disease will be determined by qualitative observations and quantitative trials. The researcher will study parkinson disease symptoms, treatments and the causes.There are still many questions raised on how the disease is developed and ways you can prevent it. Researcher will be using the before and after study design to gather information about the disease. The first experiment for gene therapy with a subject suffering from parkinson’s disease were unsuccessful compared to experiments now. Information will be gathered about a group of subjects that use old treatments such as the levodopa medication or that has experienced deep brain stimulation will be conducted as data. It will also be observed how the patients quality of life has been after receiving those medications. Most importantly it will be observed how old medications help with the production of dopamine cells. After gathering that information there were will be subjects with parkinson’s disease that have tried the gene therapy experiment and others that have not. Last, results of dopamine production with patients receiving the gene therapy will be compared between two subjects.. From data collection it will be concluded if gene therapy is a progressing treatment that will become an alternative for adults suffering from Parkinson’s disease.DiscussionThe study will not only make an amazing impact for the science of disease prevention, but to the 1% of adults that suffer from Parkinsons day to day. They won’t have to intake medications that give them outrageous side effects like suicidal effects. This experiment will be tremendous growth and become an alternative to millions of people. These people are being limited to having the option of taking medication or surgery. If your disease is at its final stages then it’s believed you are no longer able to be saved from the nightmare that is parkinson’s disease. The hardest stage to tackle in this disease is its final stages. There were many experiment done that were unsuccessful and caused hormonal changes with subjects. Scientist were realized that depending on the patients case is the way to determine how to handle treatment. For example, a patient in their final stage of parkinson may need a higher dosage then a person recently diagnosed.The research in this study will give you a better understanding of why this alternative will work. The background information and understanding of the disease is what will help succeed with solutions. If the disease is not well known and studies then it’s impossible to target the cells that will put it in remission. This isn’t the only risk that will follow, there are also hormonal side effects to this experiment that need to be corrected. There is still much more research to be conducted in order to make gene therapy a reliable treatment. This experiment has made amazing breakthroughs and its the path to discovering the cause of the disease and how to completely put it in remission.Limitations/ChallengesThe subjects in this experiment are of ages 60 years and older. About 60,000 people in the United states have the disease in high hopes of receiving a permanent result. The amount of money it takes researchers to perform this experiment on each person is very costly. It can be up to $400,000 to successfully accomplish a gene therapy experiment. Furthermore, a limitation is that doctors to this day are not able to determine the cause of the disease. Relatively there is no way of finding a solution for something you don’t know the cause. Parkinson’s has too many factors that make it hard to settle on one theory that can be the cause. There are so many routes you can take with solving the mystery of the Parkinson’s disease predicament. Last, the experiment takes time and has a different outcome for each patient. Data collection was gathered via secondary sources. The researcher had to conclude their hypothesis with experiment conducted by other researchers. It was a challenge not being able to have your own result and arrive at a conclusion with your own findings.ConclusionTo conclude, the results from the gene therapy experiment prove it is a great alternative for people suffering from Parkinson Disease. Gene therapy has been around for decades and has had successful results that remain unrecognized. The cost of the treatment is not convenient for the people that are not able to afford it but is completely worth it. Scientist are still trying to find a solution that will remain permanent, but as of now it is safe and effective. The clinical trials conducted on subjects of ages 60 years or older concluded that the dopamine deficiency cause in patient have increased. This dopamine enhancement gradually improved troublesome symptoms like tremors in the volunteers . A progression of treatment like this will completely put, not only Parkinson Disease, but other gene deficiency diseases. The people that suffer with the disease day to day now have high hopes of living a stress free life.