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Gene Therapy
The treatment of genetic diseases has appeared a daunting challenge because there seemed little to be done if the immutable basic blueprints of the body have a serious imperfection (Beutler Pp). Even fifty years ago it was possible to greatly improve the quality of life and to actually "save the lives of patients with some such genetic diseases" (Beutler Pp). Success approaches included dietary manipulation as in phenylketonuria or galactosemia, surgery to correct various deformities, and often avoidance of inciting factors in the environment, "as in acute intermittent porphyria" (Beutler Pp). However, until the latter part of the twentieth century, the possibility of actually changing the faulty genetic blueprints was beyond the imagination of the realistic medical scientist (Beutler Pp).
Today it is certain that success in gene therapy will require the application of various technique that are tailored to the disease that is being approached (Beutler Pp). The majority of hereditary disorders will require approaches that "result in prolonged expression of the transgene, although in some storage diseases, particularly Gancher disease, transient expression...
The degree of control and the range of tissue expression will vary from application to application, thus, the design of appropriate gene therapy will require understanding not only of the molecular biology of the gene that is being transferred, but of the pathogenesis and clinical course of the disease that is being treated as well (Beutler Pp).
Gene therapy is the fusion of medicine and a variety of scientific fields, including cell biology, molecular biology, and immunology and is subject to all the rigorous standards and guidelines of each of these fields (Beutler Pp). Both viral and nonviral strategies for the delivery of exogenous genetic material…
Work Cited
Beutler, Ernest. "Gene therapy of human disease. (Reviews in Molecular
Medicine)." Medicine. 1/1/2002; Pp.
When these cells behave in an abnormal manner, it can lead to acute myeloid leukemia. Various gene therapy-based tests have been conducted on the guinea pigs as model animals. The gene therapy-based experiments have shown that deafness can be cured with the help of gene therapy. One of the main reasons of the deafness is the destruction of hair cells in the cochlea. A gene that has been known
gene therapy. The writer looks at its history, its use and the possible problems it presents. There were four sources used to complete this paper. Medical science has done amazing things in recent years. Through the use of research the average lifespan is longer than ever before, and the quality of life continues to rise. Recently it seems there is nothing medical science cannot make happen. Today the world stands
Gene Therapy FDA Ethics can be considered to come from personal values. From both a medical and a business perspective, ethics are the reasons that some news stories should be followed from beginning to end and all in between. "On Sunday morning, 23 February 1997, the world awoke to a technological advance that shook the foundations of biology and philosophy. On that day, we were introduced to Dolly, a 6-month-old lamb that
Implications for ongoing research into genetic therapies and side effects/later developments are discussed at length. Yannaki, E. & Stamatoyannopoulos, G. (2010). Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease. Annals of the New York Academy of Sciences 1202: 59-63. Though the clinical trial these two researchers are involved in does not yet have results that are ready for publication, the review of the risks
In the case of Jesse Gelsinger it was found out later that there were many rules and regulations that were in place not followed to the letter of the law like they should have been. When peoples lives are at stake it is imperative that not only are their strict rules and regulations set down but that there is a process in place to make sure that those involved
Of this there are two types: Splicsome mediated - the delivery of RNA (a Repair Method) a. Strand that pair specifically with the intron next to the mutated segment of mRNA and leaves the correct strand. b. Simultaneous delivery of a correct version of the segment to replace the mutated piece in the final RNA product. Translation of the repaired mRNA to produce the normal functional protein. Triple Helix-Forming Oligonucleotide: Oligonucleotides that bind