The Potential of Gene Therapy to Cure Diabetes
Abstract
Gene therapy treats and prevents a disease by introducing a vector of genetic material into certain cells to alter the function or ability of a gene. The promise of gene therapy as a cure for diabetes has been considered ever since this new technology emerged into the clinical and research sphere. Although such methods have yet to undergo human clinical tests, gene therapy holds much potential to bring a radical new way of treating autoimmune diseases such as diabetes. By targeting certain genes that control the insulin and ?-cell production in the pancreas, gene therapy will someday fulfill its potential to cure the disease that is the number one cause of heart disease in the
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(Bottino et al., 2003).
The Potential of Gene Therapy for Diabetes 2
There are two types of diabetes: Type I and Type II that operate in slightly different manners. Type II is a result of an overload of insulin in the body, causing the immune system to attack the ?-cells whereas Type I is a result of an inability to produce insulin. Type I Diabetes is a chronic disease that is usually diagnosed in childhood and has a proven hereditary and genetic link. (ADA, 2005). Hence, current research on gene therapy is focused on the genes that can cause Type I Diabetes. In the last decade, gene therapy has emerged as an effective and very probable means of clinical intervention for victims of this life-altering disease. Gene therapy involves the integration of manipulated genetic material such as proteins or DNA into a cell to change its function. (Rudolph et al., 1996).
An ideal gene therapy treatment, would eliminate the need for daily insulin injections or strict monitoring of blood glucose levels. Under such a protocol, insulin would be produced naturally and the body?s metabolism would be able to control the production of the valuable enzyme. Currently, there are three types of potential protocols that can treat type I diabetes.
First are anti-apoptotic procedures, where all gene therapy research on diabetes will hopefully someday reach. These aim to prevent Type I Diabetes before it sets in and stop the
Diabetes is a very common chronic medial disorder and expected to be a big medical challenge of the twenty first century (Clark, 2004). It is a condition, in which the glucose level in the blood becomes so high that the body is unable to utilize it properly. This long-term condition results when the pancreas of the victim is either not able to produce enough insulin (sometimes even cannot produce any insulin) or the insulin produced cannot work properly. Diabetes is divided into two main types: Type 1 and Type 2. Type 1, also known as insulin dependent diabetes milletus (Masharani, 2008) cannot be prevent while Type 2 can be prevented but if the patient is at pre-diabetes stage.
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.
Would you consider altering your DNA if it could save your life? Scientist have been working on gene therapy since the 1970's, this biotechnological form of medicine is the attempt to medically modify cells to help eliminate or prevent diseases by correcting defective genes. Imagine the possibilities of having your DNA tested for heritable diseases and being able to eliminate such diseases from your future.
Diabetes has being around for many centuries and it’s still a current problem in our society. There is two types of diabetes type I and type II. Type I diabetes is an autoimmune disease and the usual treatment is insulin. With type I diabetes the pancreas doesn’t produce insulin a hormone that is necessary for the body; it’s important since it acts like a taxi because it facilitates the ride of sugar (glucose). Glucose is important to the cells because they rely on glucose for energy. Insulin helps the body to balance the glucose levels: by not letting the body become hyperglycemic (high sugar levels) or
The Diabetes Treatment written by Sheriff Sheik Abdulazeez discusses the major health concern of diabetes. This is a deadly disease; it accounts for 5% of deaths around the world annually. They are two types of diabetes. Type 1 diabetes is characterized by the immune complex mediated attack on insulin produced by B cells in the pancreas, and type 2 diabetes arises due to inability of insulin to regulate glucose in the blood. The treatment previously used for diabetes was inadequate because it did not treat the causes of the disease. With the stem cells, scientists have greater chances of finding a better solution to treat the disease. This can be accomplished by using adult, Pancreatic, and embryonic stem cells to produce insulin secreting cells.
My family has a long history of Type 1 Diabetes. One thing that has made me interested in biomedical engineering is how far the treatment of diabetes has come. When my grandfather was diagnosed, there was little anyone could do to treat it other than to tell him to take insulin shots around the time when he ate his meals. He did his best, but in 2008, he died of kidney failure due to the strain that diabetes had put on his body over his lifetime. However, when my cousin was diagnosed with Type 1 Diabetes in the early 2000’s, there was new technology like the insulin pump which took almost all of the human error out of the equation. I find it amazing that in one generation, the life expectancy of someone with Type 1 Diabetes has increased to the point where it’s equal to someone without the disease at all. It makes me wonder if during my lifetime someone will create the technology that makes Type 1 Diabetes a disease of the past.
With our current society rapidly advancing in both the technological and medicinal world, many new treatments have been born that can be used to treat genetic conditions that regular antibiotics and surgeries simply have no positive or progressive effect. Gene therapy is when a newly developed gene is transferred, or introduced, to an already existing gene, it is the attempt to treat one’s genetic disorder at the molecular level and could significantly improve the human population and provides hope to people with disabilities. Gene therapy goes straight to the source and alleviates symptoms of the disorder. The gene receiving this new development is in some shape or form, mutated, or defective. If the gene therapy is a
Several people in the United States suffer from diabetes. Some of the worst cases can cause kidney failure and even death. Type 1 diabetes is an autoimmune metabolic condition which kills off all the pancreatic beta cells that produce the insulin needed for glucose regulation in the body. (2) Finding a cure for type 1 diabetes would be one of the most successful thing we can do. By testing a human insulin producing cell, we can see if injecting a type 1 diabetic with the insulin producing cell that would then help generate their own insulin producing cells.
Deoxyribonucleic acid (DNA) was discovered in 1944 by Avery and colleagues. Avery identified DNA as the primary genetic material. Watson and Crick later discovered the double helix structure of DNA. Leder and co-workers deciphered the triple nucleotide code that designated the amino acids from which proteins were built. The science of molecular biology was born (Sokol, Gewirtz, 1996). In 1990 a four year old girl who was suffering from severe combined immunodeficiency (SCID) was the first to undergo gene therapy. White blood cells were removed from the girl and the cells were inserted with normal copies of the defective gene and returned into the girls circulation. Her condition improved with four treatments and
Gene therapy is a technique that allows for the modification of genes to treat or prevent disease and may become a viable way for doctors to treat a disorder without the need of drugs or surgery. This technology is still experimental, being only about three decades old with the first successful human trial occurring in 1990 when Dr. William French Anderson treated Ashi DeSilva for Adenosine deaminase deficiency (ADA deficiency), which is a genetic, metabolic disorder which causes immunodeficiency. Researchers are testing several approaches to gene therapy, which include replacing mutated, disease-causing genes with a healthy copy of the gene, inactivation a malfunctioning gene, and introducing new genes into the body that will help fight diseases. Over the last two decades, about 2,000 trials have been conducted or approved and future prospects for this technique are encouraging, include the engineering of human genetics in ways that may prove controversial and difficult to predict. This paper will explain how gene therapy works, what it has accomplished so far, its future and current hurdles, and what this all means for our species.
A novel therapeutic modality, gene therapy is the transfer of nucleic acids – DNA or RNA – into select somatic (body) cells to correct genetic defects or produce therapeutic proteins. It can be performed ex vivo (“out of the living”) or in vivo (“within the living”). In the ex vivo method, the target cells are removed from the patient, genetically modified, and reintroduced into the patient's body. This approach is efficient but limited to easily accessible cells such as epithelial cells (cells covering internal and external surfaces of the body), muscle cells, blood cells, and stem cells. In the in vivo method, a gene delivery system transfers the genetic material into the target cells within the patient's body. The development of safe
There are over three million Americans that have already been diagnosed with type 1 diabetes, and another 15,000 children and 15,000 adults being diagnosed each year. Although frequently overshadowed by the better known type 2 diabetes, type 1 diabetes is an autoimmune disease that is not caused by “being fat” or “eating unhealthy” as many would ignorantly infer. Type 1 diabetes, however, is caused by the attacking of the immune system on the pancreas, denying it the ability to carry out its most important function, producing insulin to help control one’s blood glucose levels. For over a decade now, biomedical engineers, scientists, and doctors have been working diligently and effectively on creating a closed-loop
We carry about a thousand genes in us. Most of the genes we inherit come from our parents. Hundreds of genes can contribute to common conditions like breast cancer, Alzheimer’s disease, colon cancer, and rare disorders like cystic fibrosis and muscular dystrophy (Harvard Women’s Health Watch, 1998). Gene therapy is known to treat genetic diseases and other health problems. Figure 1 illustrates how gene therapy works. Genes are introduced into a human’s body by replacing the bad cell with a new cell. In Katherine Hunt’s article, (Gene Therapy), it explains the history of gene therapy that took place between 1970’s through 1990’s. In the 1970’s, scientists came up with “gene surgery,” to treat inherit diseases that are caused by bad genes. In
Every living thing is the product of the genes that were passed down from ancestors. Genes make up everything we are. One gets their traits from their parents. Most people live full lives with relatively good health. However, some people inherit mutated genes or faulty genes. This could lead to genetic disorders that could be life threatening. Even today, many genetic disorders still remain incurable, leaving many people without hope. Genetic therapy could be their answer. It is through this research that the cure for genetic disorders can be found. Though some people believe it is unethical or immoral to alter genes, current therapeutics have not been able to save the lives of the patients with these diseases. Genetic therapy
revolutionizing medicine and treating disease. It is believed that Gene Therapy holds the key to