Gene Therapy Research CER

Gene Therapy Research CER

By Rita Debbaneh

     Genes are hereditary units which are passed down from the parent to the offspring through reproduction. They're found in DNA strands, which are made out of a sugar-phosphate backbone and nucleotides bound by hydrogen bonds. DNA strands can be found in chromosomes, which are found in the nucleus of a cell. When there is a defect in the DNA, it is known as a mutation. Several examples include insertion, when a gene is inserted in the chromosome, and deletion, or when a gene is deleted. Mutations can often be harmful and cause diseases or faulty proteins. However, there is a cure in the making, called gene therapy.

           Gene therapy is when a faulty gene is replaced, in order to cure or prevent an illness. The cell will not accept a new gene inserted directly, so a carrier known as a vector is genetically engineered to do so. At times, a weakend virus can be used, because they can longer cause disease. The vector is usually injected or given through IV (through the veins).

           

          The U.S. government should allow research for gene therapy to commence, and if already started, to continue. There are numerous regulations and laws to protect people acting as research subjects or seeking gene therapy. The U.S. Food and Drug Adminstration, or the FDA, oversees all cases of gene therapy projects, and can easily turn down anything which looks suspicious or hazardous. Additionally, the National Institutes of Health, or NIH, provides a strict protocol for scientists and institutions to follow when completing research for gene therapy, while the Institutional Review Board (IRB) and Institutional Biosafety Committee (IBC) review and approve gene therapy trials. Furthermore, defective cells will be replaced, eradicating many diseases, such as Alzheimer's and Parkinson's disease. With the plethora of departments and institutions closely monitoring clinical trials and resarch of gene therapy, as well as the many regulations, any health hazards can be avoided. If research can continue, a better understanding of gene therapy may come to light to the public. Also, at first, only people with a higher income and per capita may afford this procedure, but as more clinics for gene therapy are made, and more efficient ways are discovered, they can be open to people with lower income as well, erasing the ethical issue of "class." Many lethal diseases can be prevented, and even though this treatment can cause hazards like inflammation, it must be considered that many surgeries also have these downsides, and this is a surgical operation which can stop many sicknesses. 

Links

Videos 

https://m.youtube.com/watch?v=U3RygvuSrok

https://m.youtube.com/watch?v=Ez560GnkSrE

Additional Reading

https://www.ama-assn.org/ama/pub/physician-resources/medical-science/genetics-molecular-medicine/current-topics/gene-therapy.page?

 http://learn.genetics.utah.edu/content/genetherapy/gtsuccess/

Important Vocab

Gene Therapy: When a faulty gene is replaced to prevent illness.

IRB: Institutional Review Board.

NIH: National Institutes of Health.

IBC: Institutional Biosafety Committee.

FDA: Food and Drug Adminstration.