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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 had been cloned directly from a single cell taken from the breast tissue of an adult donor." (Brannigan 10) This report is not about cloning per se, but it is about the underlying industry for gene therapy and the Food and Drug Administration regulations as they pertain. The secondary goal is to point out who controls the legal aspects of a new drug application process. Like cloning, gene therapy is in some circles a great medical and scientific breakthrough while in other circles it is the be all and end all of the human race. That is why it is wise to understand not just the technology, but it is also prudent to see who regulates any new technology like gene therapy.
"Since the issuance of the "Points to Consider (PTC) in Human Somatic Cell Therapy and Gene Therapy" in 1991, the range of gene therapy proposals has expanded to include additional classes of vectors and use of vectors in vivo via direct vector administration to patients. This guidance document...
These guidance's are not regulations, but rather represent issues that the Center for Biologics Evaluation and Research (CBER) staff believes should be considered at this time." (Murphy)
Gene therapy is a surprisingly simply process where genes are manually inserted into an individual's cells and tissues with the intention of treating any inherent diseases. The methodology shows promise in several areas but currently has seen more success in the treatment of known hereditary diseases. Current research has both the business and governmental sectors vying for breakthroughs in areas like cancer, cystic fibrosis, heart disease, hemophilia, wounds, infectious diseases such as AIDS, and graft-versus-host disease. This is a relatively new industry and the growth potential is very high. The key with gene therapy is that new discoveries are made not annually but daily. This process has already made its way into the daily options of mainstream medicine for physicians dealing with certain viable diseases and conditions. There are some who still feel that the methodology is too controversial,…
References
Brannigan, Michael C. Ethical Issues in Human Cloning: Cross-Disciplinary Perspectives. New York, NY: Seven Bridges Press, LLC, 2000.
Food and Drug Administration. "Cellular & Gene Therapy Products." Retrieved on December 7, 2009, from FDA at: http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/default.htm
Murphy, Dano B.. "Guidance for Industry: Guidance for Human Somatic Cell, Therapy and Gene Therapy." U.S. Department of Health and Human Services, Food and Drug, Center for Biologics Evaluation and Research Administration. March 1998.
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
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
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
Administrative Law REGULATING STEM CELL THERAPY Administrative Agency: Food and Drug Administration Article 1 Section 1 of the federal Constitution creates administrative agencies, which are law-making entities but with limited powers (USLegal, 2014). The rules and regulations created by an administrative agency are lawfully enforceable. It assists in the faster management of cases and is, therefore, a big help to U.S. courts. The administrative process is also a valuable resource for other important
" The Ecumenical Review, 54. Kneen looks at agricultural genetic engineering and takes the view that because agricultural genetic engineering may help solve food shortage problems as well as enhance the environment, such activities should be looked at positively. Kneen puts good arguments together to make his points, but also writes from a biased viewpoint, so his article should be used in conjunction with other writers who are not quite so
The exchange of the defective gene could be brought about by a process called homologous recombination. One of the first gene therapy experiments, though well intentioned, caused the death of the patient. Researchers at the University of Pennsylvania introduced a normal gene into a boy's body by using the rhino virus as a carrier. The body's immune system attacked this as a pathogen. This led to eventual organ failure