2.1. Gene Therapy
Gene therapy involves the introduction of genetic material into cells in order to treat or prevent disease. Classical gene therapy has been described as “using DNA as a drug”, in which DNA carrying genes is transferred into cells by artificial means. After decades of research, this approach has now been successfully used to treat a number of conditions in humans. This section presents the historical background to the development of gene therapy.
Genes as theoretical units of inheritance were first described around 1900 by the botanists Erich von Tschermak, Hugo de Vries, and Carl Correns based on their observations of phenotypic segregation ratios in plants. In 1911, Thomas Hunt
Morgan’s studies of sex-linked traits in fruit flies led him to suggest that the genes responsible were carried on the sex chromosomes and that all genes were associated with particular chromosomes.
Although the physical location of genes was known to be chromosomes, for decades the biochemical nature of genes was controversial. Chromosomal proteins were a leading candidate owing to their complex chemical compositions. The problem was solved by Oswald Avery in 1944 when he isolated a chemical ‘transforming principle’ able to stably transform a strain of Pneumococcus Type II (small, rough colonies) into
Pneumococcus Type III (large, smooth colonies) (2). He identified the transforming principle as “sodium desoxyribonucleate”, or in modern nomenclature, DNA. Avery’s work was later
Genes can either be sex-linked or autosomal. If a gene appears mostly in one sex chances are the gene is sex-linked and if it appears frequently in both sexes it is most likely autosomal. Using Drosophila melanogaster, also known as the fruit fly, we will determine whether the gene is sex-linked or autosomal. Drosophila melanogasters have a relatively short life span and are an excellent organism for genetic studies because it has simple food requirements, occupies little space, is hardy, completes its life cycle in about 12 days at room temperature, produces large numbers of offspring, can be immobilized readily for examination and
Imagine the possibility of eliminating serious genetic diseases from the world. Imagine the idea of treating, preventing or even curing diseases that are yet to be cured. Imagine the feeling of being given improved health and a prolonged lifespan. This can all be accomplished with the aide of genetic engineering. Human genetic engineering refers to the process of directly manipulating human DNA to produce wanted results. DNA is a simple but very complex chemical that has the power to change the world and has begun to do so already. Many opponents to gene therapy fail to realize that genetic engineering has great potential to become very important in the biomedical industry. Though controversy exists regarding the ethics of human genetic engineering, it can produce numerous benefits, which outweigh its disadvantages and side effects; therefore, scientists should be able to manipulate the human genome for the purpose of helping people with serious medical conditions.
Genetics is the study of genes, heredity and variation in living organism. It is important in understanding how heritable traits are passed down from one generation to another. Here in this study, the identification of a particular mutation was explored by discovering how it is transmitted and where it is located. Drosophila melanogaster, a species of fruit flies, was the model used to illustrate the process of gene inheritance. Virgin female fruit flies were collected over a span of two- three weeks and crossed with balancer chromosome male fruit flies. The progeny, also known as the F1 generation, was then used to perform a test cross. The test cross was done between an F1 male and a virgin female from the parental generation. The results of the test cross, determined the mutation as well as which chromosome it was located on.
Mutant fly’s names are given most of the time by their mutation. One way of observation Morgan used was the chi square. A chi square (X2) statistic is used to investigate whether observed and expected ratios differ due to chance. He used this to see if the ratio he expected was going to be the same one that he will observe. The importance of Morgan's earlier work with fruit flies was that it demonstrated that the associations known as coupling and repulsion, discovered by English workers in 1909 and 1910 using the Sweet Pea, are in reality the obverse and reverse of the same phenomenon, which was later called linkage. Morgan's first papers dealt with the demonstration of sex linkage of the gene for white eyes in the fly, the male fly being heterogametic. His work also showed that very large progenies or a descendant of Drosophila could be bred. His work also demonstrated the important fact that spontaneous mutations frequently appeared in the cultures of the
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.
Gene doping is an extension of gene therapy. Though, instead of injecting DNA into specific cells for the purpose of repairing the function to a damaged or missing gene, gene doping involves implanting DNA for the purpose of enhancing athletic performance. The World Anti-Doping Agency (WADA), an international organization created in 1999 to "promote, coordinate, and monitor the fight against doping in sport in all its forms," defines gene doping as the "nontherapeutic use of cells, genes, genetic elements, or modulation of gene expression, having the capacity to enhance performance" (World Anti-Doping Agency, 2008).
A second clinical trial evaluated the use of a pMFG gammaretroviral vector with a gibbon ape leukemia virus envelope for gene therapy of patients with X-SCID. The ten patients enrolled in this trial did not have HLA matches for a HSCT and did not require chemotherapy priming prior to gene therapy. At the end of 8-9 years of follow-up, all patients had CD34+ T cells that seemed to be in proportion to the number of T cells transfused. All patients had detectable CD3+ T cells and 60% of these patients had normal levels within six months after therapy. 90% of patients had CD4+ T cells within normal range, 60% had CD8+ T cell populations within the normal range. There was an initial increase in the level of NK cells and B cell recombination
Cancer occurrs by the production of multiple mutations in a single cell that causes it to proliferate out of control. Cancer cells often different from their normal neighbors by a host of specific phenotypic changes, such as rapid division rate, invasion of new cellular territories, high metabolic rate, and altered shape. Some of those mutations may be transmitted from the parents through the germ line. Others arise de novo in the somatic cell lineage of a particular cell. Cancer-promoting mutations can be identified in a variety of ways. They can be cloned and studied to learn how they can be controlled.
Gene therapy is a relatively new practice in genetics that aims to correct mutations at a molecular level instead of using drugs or surgical approaches. Different approaches to gene therapy include gene augmentation therapy, gene inhibition therapy, the killing of specific cells, somatic gene therapy, and germ line gene therapy. Through gene augmentation therapy, faulty genes would be taken out of the genome sequence and replaced, effectively ending the effects that the mutated gene had on the body. An example of a gene that would be replaced is the mutated p53 gene. If functioning correctly, the p53 gene should stop the formation of tumors.It works by binding to DNA in the cell and stimulates the production of p21, which when combined with a cell division- stimulating protein, stops cell division. When the p53 is mutated, it fails to stimulate the production of p21 and the cell begins to divide uncontrollably. The gene therapy would replace the faulty p53 gene with a normal p53 gene.
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
“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.
Genes play an essential role in who we are today. They also can play a role on if somebody will develop certain diseases and illnesses. Scientists are starting to discover genetic mutations that can increase a person’s risk of cancer. For example, the BRCA gene test is type of blood test the doctors use to help identify mutations in genes that can cause a patient to be susceptible to breast cancer. Along with genetic testing for cancers, scientists and doctors are also using gene therapy as a way to treat cancers. That lead me to the question, can gene therapy be used as a way to cure certain types of cancer? This question is interesting because in 2016, there was an estimated 595,690 deaths from cancer in
The word science comes from the Latin word "to know" (Science Made Simple, 2014). In
Another man who contributed greatly to the study of genetics, was an American biologist by the name of Thomas Hunt Morgan. He studied the ways that characteristics were passed from one generation of fruit flies to the next. He learned that the genes in fruit flies behaved in the same way as the genes in pea plants. He also noticed that certain genes were inherited together more often than random chance should allow.
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