Using this process, it promotes the freedom of scientific inquiry and the freedom of knowledge. Although this is experimental, medical professionals generally use the best treatment methods available. It can also prevent the onset of devastating illnesses. Summarize the mechanisms, risks, and potential benefits of gene therapy; Identify ethical issues involving gene therapy and the regulatory agencies that provide oversight for clinical trials; Compare somatic-cell and germ-line gene therapy ; Many types of genetic engineering have yielded clear benefits with few apparent risks. In this blog, I’ll summarize the growth, risks, and regulatory requirements for gene therapy research. Some cancers are caused by more than one gene and some vectors, if used incorrectly, can actually cause cancer or other diseases. One of the benefits of somatic gene therapy is that it can be utilized in a way that can possibly cure patients that are severely ill. This viral genetic material is armed with the correct gene. I’ll also discuss how a centralized biosafety review process can be beneficial for this type of research. The substitution of defective genes for functional copies also raises ethical questions that must be addressed before accepting these treatments to prevent cancer . Mostly, retroviruses have the capability of injecting their genetic material into the host cells. Gene Therapy Pros and Cons: Gene therapy is involved in the replacement of a defective gene within a cell’s nucleus with the genetically healthy and functional gene.It is also known as ‘Gene Augmentation‘.Genes are basically nitrogenous based codes that encode secret information of all of the amino acid formation in the body. Gene therapy is a complicated field of research and many questions remain to be answered. The long-term safety of such treatments has yet to be determined. This means that the gene therapy can reduce the levels of dysfunctional proteins that may be causing disease, introduce the DNA code required to produce a functional protein, or increase the level of production of proteins required for disease-response. What are the risks and benefits of gene therapy? One of the potential benefits of gene therapy is that it can be used to treat diseases with single or few administrations rather than frequent dosing. Ok, so how do we arrive at the numbers for this ratio. With the exception of Luxturna which has been FDA approved, doctors are still experimenting with gene therapy. Therefore, an appropriate drug price will reflect a 1:1 cost-effective ratio. The therapy depends upon vectors, which refer to the carriers of the normal genes that transfer them to the cells that have the defective gene. Reciprocally, if a gene therapy delivers meager health benefits, then the price should be low. Source. Viruses are one of the most popular vectors. Introduction. That is the essence of a cost-effective approach to drug pricing. Cost is fairly straightforward, it’s the price of the drug. Some gene therapies appear to be effective in curing certain conditions. The possibility of gene therapy began in the 1960s and 1970s when scientists discovered the first evidence for uptake and expression of exogenous DNA in mammalian cells. Gene therapy research is beginning to boom in the clinical setting.