Biogenetic Therapy - Essay Example

BIOTECHNOLOGY/BIOLOGY BIOTECHNOLOGY WEEK 3 DISCUSSION Transgenic proteins are produced within a multi cellular host system like in transgenic animal or a plant, where a foreign gene has been deliberately introduced into its genome to produce the relevant proteins, while recombinant protein production occurs through cloned DNA sequences at the cell level, which normally encode an enzyme or a protein whose functions are known. The medically important coagulation factors VII and IX are examples of human proteins produced transgenically. (1) 2. Gene therapy is still in the experimental stages and involves the insertion of the genetic material DNA or RNA into the relevant cells to provide the absent function or to provide the cells with a new function to treat diseases with a genetic origin. In contrast protein therapy involves employing clearly defined and precisely structured proteins to manipulate malfunctioning cells towards healthy function. Using the principles involved in protein therapy actual human DNA is introduced into a host E. coli cell, which are then encouraged to grow and multiply in a normal manner. The inserted human DNA in these cells causes the production of recombinant human insulin by these cells. Using gene therapy it might become possible to engineer the duodenum cells into become beta cells to produce insulin, thereby treat the disease. Using gene therapy it may also become possible to address the issue of autoimmune response against the beta cells in Type 1 diabetes, thereby providing a cure for it. (1) 3. From a molecular biology perspective the common factor in Sickle-Cell disease, Cystic Fibrosis, and Huntington’s disease is that alterations in the genetic material are responsible for faulty protein production. In the case of Sickle-Cell disease a single base-pair change in the gene that encodes a part of the hemoglobin protein is responsible for the disease, while in the case of Cystic Fibrosis the commonly occurring cause is the a three-base pair deletion in the protein involved in chloride transport into and out of the cells, and in the case of Huntington’s disease, it is the enhanced number of C-A-G repeats at the beginning of the Huntington gene that codes for an excess quantity of glutamines in the Huntington protein. (1) 4. It is more or less an accepted that in many diseases it is the combination of environmental and genetic factors that lie behind it, one in support of the other. Extending this combination of contributory factors to all the events that occur in the environment may not be plausible, but in the case of motor vehicle accidents genetic factors may be a possible contributory factor for cause of the accident. Let us take the case of patients suffering from narcolepsy for which there is increasing evidence of a genetic cause. People suffering from narcolepsy are prone to severe sleep attacks even when they are active, and thus are more prone to motor vehicle accidents. (2). We observe that some individuals are more prone to accidents than others in any given environment, and this suggests that there may be a genetic basis for this proneness to accidents. 5. The essential goals that were ascribed to the Human Genome Project (HGP) involved the providing of a full and precise sequence of the 3 billion DNA pairs that constitute the constitute the human genome and the finding of all of the nearly 25,000 human genes; sequencing the genomes of other organisms like the fruit fly and the mouse that are significant for medical research; development of new tools to analyze the obtained data; and the evaluation of the impact of the advances in genetics on individuals and society. (3) Clear diagnosis of the presence of a disease and the exact status of the disease in a patient is essential for the efficient and effective management of the disease in the patient. Current diagnostic procedures are not totally satisfactory for this aspect. The better understanding of disease processes through proteomics removes this deficiency in diagnosis. For example proteomics would permit the use of markers to define different types of tumors, to make for a more definite diagnosis and prognosis of the disease in an individual thus indicating the preferred treatment for managing the disease. Again the use of proteomics for the analysis of body fluids and tissue biopsies offers a more definite diagnosis of the disease and its prognosis in an individual than current laboratory tests offer. The prognosis of some diseases is unique in individuals. Proteomics allows the identification of this unique progression of the disease and the right intervention treatment strategies, as is the case of metastasis. In the treatment aspect proteomics offers the means to the creation of more effective medications. For example more specific and effective antimicrobials are possible, with the use of the better understanding of proteomics. Random screening is the current means employed by scientists find growth inhibitors of pathogens. Microbial genomics allow for a more rational approach for the development of more powerful and effective antimicrobials. (4) 6. The current line of thought is that only about two percent of the human genome actually contains protein-related information. As out understanding of the human genome has grown, so has the realization that the human genome contains less DNA that encodes proteins and more introns and intergenic DNA. A large portion of the DNA as per our current understanding has no role to play in the gene structure or gene functions. However, as the understanding of genomes and the control of gene expression has developed, more and more functions for the DNA have been found. This suggests that our understanding of genomes and the control of gene expression is currently limited and that as this understanding develops even further we are likely to find the DNA involved in more functions to take up the large portion of DNA for which there is no structural or functional roles at present. (5). BIOTECHNOLOGY WEEK 4 DISCUSSION 1. Bionanotechnology provides potential benefits that help us to overcome some of the deficiencies in the manner in which we do things currently. Our current disease diagnostic means are not as efficient as we would like it to be in diagnosis of several diseases like cancer. Nanotechnology offers the benefit of reaching areas where currently we cannot and through that offers the potential of early and more efficient diagnosis of diseases like cancer. Another potential benefit is in drug delivery systems, whereby drugs can be delivered in such manner that they identify the contaminated cells and deliver the drugs to these cells only. Bionanotechnology also offers the potential to develop regenerative medicines that assist the natural healing processes to work faster, thereby re-growing or repairing tissues faster. (5). However, there are concerns associated with bionanotechnology. These concerns include the possibility of opening a Pandora’s Box in bionanotechnology, where we will not only reap the benefits, but also the harm with our inability to control the course of development. Another issue is the possibility of bionanotechnology enabling the richer nations to get richer, without really allowing the poorer nations to participate in it. Yet another is that through the potential of extending the span of life with bionanotechnology will be converting humans into machines. Individual privacy is another contentious issue. The final concern with bionanotechnology is there is hardly any awareness of the impact of it on the environment and hence concern on its possible harmful effect on the environment. (6). 2. A plant or animal into which a cloned gene is successfully introduced is called a genetically modified organism (GMO). The concerns on GM foods stem from ethical and moral issues associated with genomics in the form of man playing God. In addition there are concerns that GM foods could have a negative impact on human health and the environment. Genetically modified plants used as foods offer advantages on two fronts namely as improved nutrient sources and a delivery system for vaccines. For example the gene that codes for the synthesis of beta carotene, the precursor for Vitamin A has been introduced into a variety of rice to enrich the presence of beta carotene in the rice. In a similar manner rice has also been genetically engineered to increase the iron content in it. These different varieties of rice can be grown in regions where the people have a high incidence of Vitamin A deficiency or suffer from anemia to reduce its incidence. In a similar manner plants used as foods can be genetically engineered to provide particular additional nutritional value to make up for deficient nutrition making for a healthier world. Genetically modified plants can also be used as vehicles for edible vaccines. This possibility comes from the success researchers have had with genetically engineered potatoes producing proteins of certain disease-causing microbes and the immune system demonstrating response to these proteins. Potatoes have been used, because of the relative ease with which they can be genetically engineered. Potatoes are not eaten raw but cooked and cooking has the potential to destroy these proteins. The challenge lies in genetically modifying foods that are eaten raw to produce the relevant immunity producing proteins. (1). 3. Dramatic advances in the field of science and technology, particularly biotechnology has brought into focus Intellectual Property (IP) rights and the controversy around it. Intellectual Property rights are necessary on one side to foster innovation towards further advances, but rigid IP rights on the other hand curb competition and the benefits derived from competition. Without IP rights any competitor could copy the development of the original developer, like a novel drug developed using RNAi techniques, and enter the market with this drug without having to go through the effort and expenditure involved in the development, preventing the original developer from realizing the cost of the effort and expenditure on the development of the drug. IP rights prevent such an eventuality by giving the patent holder exclusive rights for twenty years for an innovative idea making it an important factor in the development of new drugs. Severable innovations of a novel idea or invention can be used by licensed companies for follow-on innovations in the creation of new products, but non-severable improvements cannot be used by companies as it infringes on the IP rights of the patent holder. Thus when presented with new ideas companies evaluate it for novelty that does not infringe on the patent rights another company and prefer to keep out of development of that idea onto new products if it infringes on the OP rights of any patent holder. (7). 4. RNAi is a novel method for the regulation of gene expression that employs a few double stranded RNA (dsRNA) molecules per cell for this purpose. The dsRNA is obtained from the interaction between virus and transposon in natural RNAi processing. An enzyme complex known as DICER is used to cut the dsRNA into fragments. These fragments called small interfering RNAs or siRNAs become the templates for RNAi inducing silencing complex, which through the destruction of homologous messaging suppresses gene expression. (8). Yes I agree that RNAi is one of the hottest areas in current biotechnology. The reasoning for this is that the Nobel Assembly at Karolinska Institute awarded its Prize in 2006 for Physiology or Medicine to the discoverers of RNA interference Andrew Fire and Craig Mello and observed that RNAi has already found widespread use in basic science to study the function of genes, which may well lead to the development of new drugs in the future. This observation has held out with nearly six new therapies based on RNAi in different phases of clinical trials and several more in the pipeline. (9) Conventional drugs are seldom target specific, and as such are less effective and cause side effects. RNAi with its specific gene regulation capability is capable of providing a means to develop target specific drugs to degrade the intended cellular targets and hence the potential for more effective drugs with minimal side effects in comparison to conventional drugs. Novel drugs based on RNAi in the clinical trial phase include bevasiranib for treating age-related (wet) macular degeneration; ALN-RSV01 an antiviral drug that targets the nucleocapsid gene of respiratory syncytial virus; and nucleonics a plasmid based expression strategy that targets four different regions of the Hepatitis B virus. (9). Works Cited 1. Nester, W. Eugene, Anderson, G. Denise, Roberts, C. Evans, Pearsall, N. Nancy & Nester, T. Martha. MICROBIOLOGY A HUMAN PERSPECTIVE. Fourth Edition. Boston: McGraw-Hill Higher Education, 2004. 2. Stone, Kathy. “RECENT INSIGHTS AND BETTER TOOLS FOR UNDERSTANDING AND DIAGNOSING NARCOLEPSY”. Neuropsychiatry Reviews 7.5. (2006). 9 Feb. 2008. . 3. “What were the goals of the Human Genome Project?” Genetics Home Reference. 2008. U.S. National Library of Medicine. 9 Feb. 2008. . 4. Hotigger, O. Michael. “Proteomics - how proteins communicate in a cell”. 2001. Veterinary Sciences Tomorrow. 9 Feb. 2008. . 5. McConkey, H. Edwin. How the Human Genome Works. Boston: John & Bartlett Publishers, 2004. 6. “Wickramasinghe, Nilmini., Choudhary, Shivani & Geisler, Eliezer. “Bionanotechnology: its applications and relevance to healthcare”. International Journal of Biomedical Engineering and Technology. 1.1. (2007): 41-58. 7. ““Intellectual property and competition policy in the biotechnology industry”. Policy Brief. 2005. OECD. . 8. Thakur, Archana. “RNA interference revolution”. Electronic Journal of Biotechnology. 10 Feb. 2008. . 9. Perkel, M. Jeffery. “THERAPEUTIC RNAi: Delivering the Future?” Life Science Technologies. 2007. Science Magazine. 10 Feb. 2008. . Read More