Gene Therapy : A Method Of Manipulating Cells At The Molecular Level

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).

In the past twenty years, many drugs have been developed to treat the disease. Although the cause of Parkinson's disease is still unknown, scientists have been developing methods of treatment and therapy. The idea is to replace dopamine in the brain, which is accomplished, to some extent, with the administration of L-Dopa. In conjunction with other drugs, L- Dopa "inhibits the enzymes that break down L-dopa in the liver, thus making a greater part of it available to the brain" (5). This treatment is very successful, but it only hinders the disease for a time and it is by no means a cure. That leaves us with stem-cells and the role they play in treatment of Parkinson's disease.

Gene therapy is an experimental technique that is used to prevent disease. For future reference, this technique may treat disorders. It may add or remove genes to get rid of a genetic disorder. Gene therapy is a heavily debated subject. There are very respectful points on both sides of this debated topic. The pro side believes that gene therapy is the best thing that is happening to our society. However, opposers of gene therapy believe that people should refrain from proceeding with this biological advancement and it is ethnically incorrect.

Tay Sach’s disease results in an accumulation of lipids in the brain’s nerve cells. The underlying cause of Tay-Sachs disease is a mutation in a gene that codes for a lytic enzyme known as Hexosaminidase A (Hex A). Tay-Sachs as well as other lipid storage diseases are incurable, but we believe that gene therapy has the potential for widespread correction of the lipid storage defect in humans by means of the cross-correction cellular pathway. The vehicle of transportation for the functional copy of the gene is a modified version of the Adenovirus. An advantage of the adenovirus is that its genome does not integrate into the host genome to transcribe its

The substantia nigra is where the neurotransmitter dopamine is made by cells. Neurons between the substantia nigra and the stratum send messages via dopamine. As the cells of the sustantia nigra deteriorate in Parkinsons so does the levels of dopamine in the brain, which is necessary for sending messages to other areas of the brain for movement control (Obeso, Rodríguez‐Oroz, Benitez‐Temino, Blesa, Guridi, Marin, & Rodriguez, 2008). The decrease in dopamine production also changes the dopamine to acetylcholine relationship in the brain, which the striatum require to function properly. While the striatum is very complex system which involves signal suppression and enhancement within the brain, the altered ratio of dopamine and acetylcholine can cause some tracts to be under suppressed and over suppressed (Obeso, Rodríguez‐Oroz, Benitez‐Temino, Blesa, Guridi, Marin, & Rodriguez, 2008). The degradation of the cells in the basal ganglia cause is unknown, but many suggest pesticide exposure, serious consecutive brain injury and possible genetic factors.

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.

Whether gene therapy should be allowed to be used in practice is currently a very controversial topic. In my opinion, I do not think that it should be allowed. Gene therapy is a technique where genetic material is inserted into a patient to replace missing or defective genes. Gene therapy can be further broken down into two categories: somatic and germline. From there, somatic gene therapy can be approached in two ways: in-vivo and ex-vivo. For the process of gene therapy to begin, the proper vector needs to be acquired. Once a vector is chosen, the vector delivers the new gene into the target cell. Then, the target cell becomes infected with the vector. Next, the vector’s genetic material is inserted into the target cell. Then finally, functional

New discoveries on the treatment of Parkinson’s disease show that dopamine neurons can treat Parkinson’s disease. Parkinson’s disease is a disorder of the nervous system, which is a progressive disease. Parkinson’s disease is marked by tremors, muscular stiffness. It mainly affects people who are either elderly or middle aged. Although Parkinson’s disease cannot be cure lab grown dopamine can assist in being able to manage the disease in an enhanced way. Although Parkinson’s disease is not curable but can be treatable it still needs more work in order to make a cure in the future. Dopamine helps regulate movement and when there is a deficiency in dopamine that results in Parkinson’s disease.

Parkinson’s disease destroys the cells in the brain that create dopamine. As levels of the chemical fall, the body is unable to move and function as it once did. Falling dopamine levels translate to increasing movement impairment. However, over time, these medications that are prescribed to patients become less efficient, and patients are no longer able to take them. Each year doctors and scientist work together to find a treatment or a possible prevention that that will help patients with Parkinson’s disease find a relief. As for future treatments, doctors are implying to treat and defeat Parkinson’s disease through medicine that proves sustained effects throughout the day, advances in deep brain stimulation, gene therapy and neural

At present there have been great strides made in the research and therapy of Parkinson’s disease. Some of the therapies which will soon be available include the transplantation of healthy dopamine-producing tissues into the brain and the use of medications to prevent dopamine producing brain cells from becoming damaged or lost. It is the understanding of what is Parkinson’s Disease that can help those who have been diagnosed with

Parkinson’s disease is affected by the degeneration of dopaminergic neurons which is responsible to produce dopamine. Dopaminergic neurons have their cell bodies in substantia nigra pars compacta (SNpc) in basal ganglia (O’Sullivan and Schmitz, 2007). Basal ganglia are a collection of interconnected gray matter nuclear masses deep within the brain”. These gray matter masses are caudate, putamen, globus pallidus, subthalamic nucleus and the substantia nigra. Basal ganglia receive its input through striatum (O’Sullivan and Schmitz, 2007).

Genetic disorders have plagued people for ages. However, recent research and new discoveries in gene therapy has brought new hope to those unfortunate individuals that are ailed by their own genetics. Gene therapy is a technique of correcting defective gene sequences specifically responsible for the disease. In 1990, the first successful clinical trial of gene therapy was initiated for adenosine deaminase deficiency (GT). Since then, the number of clinical treatments induced worldwide has increased exponentially. Gene therapies have been a part of modern medicine for almost two decades now. Only recently have scientists started to deliver on their immense promise after a string of setbacks raised doubts about the safety of manipulating human DNA. The idea of gene therapy has been around for a while, but it wasn’t until recently that this field of molecular biology has come more into the light with its advancements over time. Experimentation has been an ongoing process with gene therapy. Although, ethical issues of controversy have surrounded this topic since the beginning. But new facts on gene therapy continues to rebuttal most controversy.

There are two concepts of gene therapy. The first concept is germinal cell gene therapy which would involve the genetic modification of germ cells.

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

Gene therapy is the method of use of genes by inserting a functioning gene into body cells in order to correct the disease by inactivating altered genes with inserting a genetically modified gene into the body in place of drugs to fight disease (Verma, 1990).