Benefits Of Gene Therapy For Parkinson's Disease

The goal of this paper is to compare the utility of adult, embryonic and induced pluripotent stem cells (iPSCs) to treat Parkinson’s disease. As such several things will be assessed, dosage of stemcells, improvement in motor function, in combination with the presence of α-synuclein proteins and cell survival.

Gene therapy is a relatively new method for treatment of chronic illnesses that are hard to treat with other methods. So far, it has been considered as a possible treatment in conditions such as cancer, cystic fibrosis, AIDS, and ALS. Recently, research has been done on using gene therapy as a treatment for the symptoms of neurological and nervous system disorders, which includes Parkinson’s disease and Alzheimer’s. As with any new treatment, the main question to consider when thinking

Just in case you are a little shaky on the topic of gene therapy I will briefly explain. Gene therapy is a type of treatment used

I chose this article because it characterizes the transduction profiles of a variety of AAV vectors that cross the blood brain barrier and transduce cells throughout the CNS. I am particularly interested in this gene delivery strategy – especially as it may apply to clinical therapeutics for Huntington’s disease (HD), a genetic neurological disease that causes widespread degeneration throughout the CNS. I spent the last few years working on studies that characterized the transduction profile of AAV9 in the CNS and periphery in a mouse model of HD. The aims of these studies were to: 1) Assess whether or not AAV9 has a tropism for the specific peripheral tissues and CNS regions that are affected in HD; 2) Evaluate whether systemic delivery using an AAV9 vector would be suitable delivery strategy to deliver an RNAi construct that could reduce HD symptoms. Accordingly, by choosing this paper, I was hoping to build a better understanding of other AAV vectors (in addition to AAV9) that cross the blood brain barrier and potentially could be used for vascular delivery of an RNAi construct throughout the CNS.

There are two types of gene therapy; germline gene and somatic gene therapy. Germline gene therapy is considered to be a safer option for humans. This method makes changes to the gamete cells that are used in the reproductive process thus, the functional genes need to be inserted into the chromosomes. Somatic gene therapy is when genes are transferred to body cells by inserting vectors into a person’s body. There are three things that gene therapy can do; replace a mutated gene that causes a disease with a healthy gene, deactivate a mutated gene that is not able to function properly, or introduce a new gene into the body to prevent diseases. There has been a clinical trial in the past where over 3000 people were treated with gene therapy and the results were good however, this treatment method has serious health

Li, and Huang discuss the controversial topic of gene therapy by first providing a factual summary of gene therapy. Their introduction goes into the specifics of the types of vectors (carriers that deliver a gene to target cells) used to transfer genes in order to aide damaged proteins. Afterwards, they transition into the detailed of process of gene therapy. They start this off with naked DNA and state it is the simplest approach to nonviral transfers and the least toxic method. However, despite being the least toxic and a nonviral transfer it does not have as great of an effect as Cationic lipids. Cationic lipids have been used in various experiments but are known to be safe only when administered in small doses. Furthermore they go into

The Parkinson 's disease (PD) is the second most common neurodegenerative disorder after Alzheimer 's disease (Lang and Lozano, 1998). It affects about 1% of the population with different ethnic backgrounds throughout the world over the age of 65 (Tanner and Goldman, 1996). The aetiology of Parkinson 's disease is not well understood; however, genetic and environmental factors are thought to play a role (Checkoway and Nelson, 1999). Pathologically, PD is characterised by mitochondrial DNA dysfunction leading to degeneration of dopaminergic neurons in the substantia nigra pars compacta (Fig.1) and subsequent reduction in striatal dopamine levels (Longmore, 2014).

Parkinson’s disease is the second most common neurodegenerative disease known to humans, characterized by loss of control of the central nervous system, and has been studied extensively by the scientific community (Hering et al. 2004). One gene loci identified as PARK7 or DJ-1, has been mapped to chromosome 1p36 and contains the DJ-1 protein (Bonifati et al. 2003). Mutations on the DJ-1 gene are shown to reduce the function of this gene, subsequently leading to the early onset Parkinson’s disease (Bonifati et al. 2003).

Parkinson’s disease has been known about since ancient times. In AD 175, the physician Galen described it as “shaking palsy” in Western medical literature. It wasn’t until 1817 that a London doctor, named James Parkinson, wrote a detailed medical essay on this disease. This essay established Parkinson’s disease as a recognized medical condition. He based the essay off of six cases he had observed previously and hoped that it would encourage others to study the disease. No such research was performed until finally, sixty years after Parkinson’s essay was published, a French neurologist named Jean Charcot studied the disease and was the first to truly recognize the importance of Parkinson’s work. As a result, he named the disease after him. Much has been learned about the disease since then; however, much of it still remains a mystery. Researchers have learned that the symptoms for this disease are progressive, degenerative, and tend to occur more in older individuals. It has been found that a dopamine deficiency of the brain is the root of the matter; although, why this initially occurs is less clear. The Parkinson’s Disease Foundation was created in 1957 to assist those who are suffering and promote and fund further research. Since the 1960’s research has only progressed more and more rapidly. Even though there still is no known cure, the symptoms can now be effectively controlled and reduced in severity. Moreover, many other foundations assisting the cause have been

Gene therapy works by adding a good copy of a defective gene to the cells in need (“What is”). Gene therapy is different than traditional treatments because it goes right to the source of the problem, rather than just treating the symptoms (“What is”). Although gene therapy is a possible treatment for many medical conditions, conditions affecting many genes is not the best candidate for gene therapy (“What is”). Inserting the gene into a cell can be an extremely difficult process. To insert the gene into a cell, a virus is attached to a vector (Vachani). This vector is used as a carrier and is inserted into the body with the new gene attached. The gene then goes into the cell’s nucleus and becomes attached (Vachani). There are multiple ways to give gene therapy to people and researchers can find the most effective way. Since gene therapy is still new and being experimented with, the side effects are still unknown (Vachani). After the vector is inserted, the body may react and cause a fever, chills, nausea, and a blood pressure drop (Vachani). These side effects are usually gone after a day or two (Vachani). Although it has not happened yet, researchers are worried that the new genes could start to affect the healthy cells (Vachani). Depending on which cells it affects it could

A new form of medicine with the potential to save and improve millions of live is gene therapy, or gene replacement therapy. Gene therapy is the use of DNA as a pharmaceutical agent used to treat or cure diseases or disorders caused by defective or missing genes. There are three methods of gene therapy: replacing a mutated gene with a healthy copy of the same gene, inactivating a gene that is not functioning properly, or introducing a completely new gene to help the patient fight a disease. Gene therapy is a promising treatment option, however, the technique has yet to be perfected and is still dangerous and experimental.

Parkinson’s disease, is a loss of nigrostriatal dopaminergic neurons that is progressive and chronic throughout the body1. Parkinson’s disease is also a neurodegenerative development disorder. Scientists and researchers have yet to discover new medications to interrupt the diminishing neurons of dopaminergic. Furthermore, there are medications that are only beneficial for the alleviation of symptomatic complications in the body. There are currently no known neuroprotective therapies or programs available, due to the insufficient knowledge on the process of the death of dopaminergic neurons. The most common factors associated with Parkinson’s disease is genetics and environmental influences. The environmental factors include the following: living

The article “Will Genetic Engineering Improve People’s Lives” explains that both kinds of gene therapy can be carried out either “ex vivo,” where cells are extracted, inserted with DNA, viruses, etc, and then reinserted, or “in vivo,” in which a vector containing DNA or whatever else needs to added is inserted into the cells. New genes can also be injected without the use of vectors, however this is less common as it only works in certain tissues and requires much larger amounts of DNA (“Will”). No matter which way is picked, once the new DNA is inside the cell, it is expressed normally and begins to create new proteins. Under the two umbrella terms of ex vivo and in vivo falls three other ways to go about gene therapy: gene augmentation therapy, gene inhibition therapy, and the killing of specific cells. Gene augmentation therapy is used in specific cases where a mutation has caused a gene to stop producing a specific product, usually proteins. New DNA is added containing a functioning version of the lost gene. This kind of therapy only works if the effects of the disease have not caused long last damaging or are reversible. Gene inhibition therapy aims to introduce genes that stop expression of other genes or interfere with

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).

revolutionizing medicine and treating disease. It is believed that Gene Therapy holds the key to