Gene Therapy Outline

Genetic diseases are pathological diseases caused by the absence or alteration of one or several genes in the cells of an organism. Most genetic diseases are caused by the inheritance of an altered or missing gene from either one or both of your parents. Because the altered gene is in every cell in your body, including your stem cells, it is practically impossible to physically alter the gene. The only possible effective way that has been found is gene therapy.

Genetic disorders are present from birth, but they may not be visibly seen until a later age. Some mutations could be heritable, or from your parents genes. Some forms of cancer can be inherited form a parent. Although, in most cases, these mutations are new changes to the DNA. “All humans have the same basic set of genes”. This means that everyone has the probability that they could get a genetic disorder. The thing that makes us different is the

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.

There are many known genetic diseases in the world. There are different types such as chromosomal and monogenic. Chromosomal disorders are when someone has too many or not as many chromosome as they are suppose to have (46 total chromosomes). Chromosomal disorders can also manifest when there are structural abnormalities. Monogenic disorders are an inherited medical condition caused by a DNA abnormalities. These diseases occur all throughout the world today. Depending on what region of the world you are in depends on what the most common ones are. Cystic Fibrosis has become the most common lethal genetic disease in the United States as of 1999. Although there are treatments available, there is not a known cure, and the effects remain burdensome

Human genetics can play a major role in determining ones physical condition. One slight change in a genetic sequence can cause a disorder that can be life threating to the organism. Most of the genetic disorders are caused by recessive allele. In most cases this recessive allele is undetectable due to the disorder not being presented in the physical appearance. Hypercholesterolemia is an example of a human disorder controlled by a single gene. This human disorder causes high levels of cholesterol in an individual due to the absence of the low-density lipoproteins.

Well, it’s quite simple. Genes are within every single one of our cells that make up our bodies. Genes tell our cells how to reproduce new cells to replace damaged or worn out cells. Without genes, cells could not replicate themselves in the way that is necessary

Genetic disorders or heredity diseases are disorders that are caused by a missing gene, abnormal gene, or chromosomal characteristic (The Free Dictionary, 2007). There are to me found many genetic disorders. Klinefelter syndrome is an example of a genetic disorder. Klinefelter syndrome affects the male rather than female (“47, XXY”, 2016).

I agree with you that there was a conflict of interest which caused Dr. Wilson to continue with the testing even though a participant died from complications. Based on your explanations, I agree that sanctity of human life should outweigh the other principles. Plus, ethics demands that we never allow scientific examination to undermine the interests and inherent value of the distinctive human being who unselfishly enroll in such research.

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

Genes is the physical and functional unit of heredity which are made up of DNA. It carries the information of that determines traits that are passed on by the parents. There are two copies of genes which comes from each parents which is to be exact twenty chromosome from each parent to make up forty six chromosome in a baby. As by the

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

Genetic disorders are problem caused by one or more abnormalities in the genome, especially a condition that is present from birth. My genetic disorder is called hemochromatosis. What hemochromatosis does is it affects the body’s ability to absorb iron. Because hemochromatosis is a genetic disorder, it is passed along from generation to generation and that is the only way that it can be passed along. There is around a total of one million people in the United States has contracted the genetic disorder of Hemochromatosis. There is a multitude of symptoms that can occur in the short term and the long term.

Gene therapy has shown to be successful in the treatment of some monogenic recessive disorders affecting the haemopoietic system, such as severe combined immunodeficiency (SCID), and Wiskott-Aldrich syndrome, by semi-randomly integrating functional genes into the genome of hematopoietic stem/progenitor cells. Severe combined immunodeficiency is an X-linked inherited disease caused by the deficiency of the enzyme adenosine deaminase (ADA), which is vital for the development of lymphocytes. In the year 2000 in Paris, researchers were able to successfully treat the immune defect in infants with a life threatening X-linked severe combined immune deficiency (SCID-X) with gene therapy. Gene therapy was a very effective

The word science comes from the Latin word "to know" (Science Made Simple, 2014). In

“Although we are all members of a single species, we differ from one another in such visible traits as the color of our skin and the shape of our noses, and in biochemical factors such as our blood types and our susceptibility to certain diseases” (Sheridan College Institute of Technology and Advanced Learning, 2013, p. 34). To understand human physical development and evolution one has to understand biological anthropology as the focus on humans as biological organisms. Biological anthropologist conduct research, and form techniques of modern molecular biology to learn about human variation and how it relates to different environment humans lived in as well as their conditions.