Ok, so how do we arrive at the numbers for this ratio. The long-term safety of such treatments has yet to be determined. Although this is experimental, medical professionals generally use the best treatment methods available. Gene therapy research is beginning to boom in the clinical setting. 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. I’ll also discuss how a centralized biosafety review process can be beneficial for this type of research. Introduction. Cost is fairly straightforward, it’s the price of the drug. Using this process, it promotes the freedom of scientific inquiry and the freedom of knowledge. 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. Gene therapy is a complicated field of research and many questions remain to be answered. What are the risks and benefits of gene therapy? Source. 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. Some cancers are caused by more than one gene and some vectors, if used incorrectly, can actually cause cancer or other diseases. Some gene therapies appear to be effective in curing certain conditions. Reciprocally, if a gene therapy delivers meager health benefits, then the price should be low. 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. The substitution of defective genes for functional copies also raises ethical questions that must be addressed before accepting these treatments to prevent cancer . 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. 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. In this blog, I’ll summarize the growth, risks, and regulatory requirements for gene therapy research. 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. 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