Gene Silencing : Current Approaches And Relevance For Drug Discovery

Because of the ethical controversies that surround gene patenting, the technology is not widely seen as beneficial to society. However, gene patents are critical to the biotech industry. “Such claims protect therapeutic proteins, like human insulin; Mabs, like Herceptin®; transgenic plants, like insect-resistant corn; and diagnostic probes for genetic diseases, which are the foundation for personalized medicine” (Karny, G. (2007, April 01). In Defense of Gene Patenting | GEN Genetic Engineering & Biotechnology News - Biotech from Bench to Business. Retrieved April 02, 2017, from http://www.genengnews.com/gen-articles/in-defense-of-gene-patenting/2052). A ban on gene patenting would have a large and negative impact on gene-based medical therapies

In 1956, Francis Crick first described what he called “The central dogma of molecular biology.” This essentially describes the flow of genetic information within cells. It states that DNA is transcribed into RNA with the help of an RNA polymerase enzyme. The RNA is then translated into a protein by protein synthesis. One thing that could drastically alter the genetic information within cells is a process called gene silencing. This process regulates the gene expression of certain genes and can occur in either transcription or translation. The process has been coined RNA interference and dsRNA gene silencing (Davidson and McCray Jr. 2011). Since direct evidence of double stranded RNA’s role in gene silencing was found in 1998 by researchers Fire and Mello, this topic has been the focus of much research in areas such as biomedical research, health care, and even agriculture. Double stranded RNA has been found to play a crucial role in things such as pest control, vector borne disease prevention, crop improvement, and in the development of therapeutics for different diseases through gene silencing. Although much research has been focused on the effects of gene silencing, there is still much more needing to be done.

On September 16, 2016, Mario Capecchi, Professor of Biology at the University of Utah and also Professor of Human Genetics at the University of Utah School of Medicine came to the University of La Crosse to speak about his discoveries in gene targeting. Specifically, he talked about gene targeting in mouse models of human disease from cancer to Neuropsychiatric disorders. This topic is connected to human biology but not to anything that we have covered in Biology 105 thus far.

There are varies of gene silencing methods and most of these methods involve disabling the function of mRNA by preventing it from being translated into a protein. However different methods use different design of molecules to disrupt mRNA. The most leading method of gene silencing is RNA interference

Gene Knockout is a genetically engineered technique in which an organism is modified to carry genes that are non-functional. The non-functional gene results from the insertion of a foreign sequence that disrupts or stops the genes functionality. The gene will no longer code for the specific metabolic processes and may cause serve implications on the organism. The method of gene knock begins in a lab with a test tube of plasmid, DNA construct proceeding to cell culture. The end goal is to create transgenic animals that carry the altered gene. Gene Knockout can be passed down through future generation if the embryonic stem cells are modified and inserted into an embryo in the early stages of development.

Although gene editing tools could cause unintended irreversible mistakes, further studies and regulations could lead these tools to become successful and effective ways of medical treatment. However, these tools should be regulated and further tested before becoming a legalized treatment. The National Academy of Science comments that “Heritable germline genome editing trials must be approached with caution, but caution does not mean that they must be prohibited” (Regalado). Editing the germline and utilizing gene editing tools could be utilized for trials, but should be proceeded with cautions. As gene editing tools evolve to change diseases, scientists are eager to utilize CRISPR without considering pitfalls. Instituting more caution would

These drawbacks however and highlighting the same is not meant to sway us from our research into genome editing rather it is merely meant to make us more careful concerning this research into gene alteration. When we go beyond these drawbacks, we can be hopeful of a future where we can use this practice not just for the purpose of disease modeling but also to facilitate the discovery into human therapeutics (Musunuru, 2013). Genome editing may now have us confused regarding the future of diseases caused by mutation, but further discoveries and breakthroughs in the technology are likely to lead us to a time when this genome editing would become common practice, and we would be able to cure at least the most common genetic anomalies. Until then, though, we work with the hopes of that

Existing gene delivery systems can be broadly classified into two categories: viral and non-viral. The first gene delivery systems were viral. Viruses were the first choice for vectors in early studies of gene therapy because they have evolved to effectively transfer DNA into target cells. To produce viral vectors, the sections of the virus's genome that enable it to replicate are removed and replaced with the therapeutic gene sequence. The result is an attenuated virus capable of efficiently transferring the therapeutic gene sequence but incapable of replicating in vivo. Disadvantages of viral vectors include limited DNA-carrying capacity, restricted cell targeting, and immunogenicity. Non-viral gene delivery systems seem to be a promising alternative. Non-viral vectors consist of naked DNA in the form of plasmids (circular pieces of DNA) or oligonucleotides (short, single-stranded pieces of DNA) and complexed – “packaged” – DNA, typically with cationic (positively charged) lipids or polymers. In general, naked DNA is safer but significantly less efficient than viral vectors. Barriers to efficient non-viral gene delivery include DNA degradation, lack of cell targeting, and limited cell uptake of DNA. However, the complexation of naked DNA facilitates the process since cationic lipids and polymers protect the DNA and facilitate transport. Current research efforts primarily focus on the development of more efficient non-viral gene

Genetic engineering is the gateway into an advanced realm of medicine. The first leap in this progression has already been established: pre-implantation genetic diagnosis, PGD. Genetic diseases can be halted by utilizing this process, particularly those resulting from single gene defects. This includes cystic fibrosis, sickle cell anemia, and Huntington’s disease. Moreover, it has recently been expanded to prevent the development of breast and ovarian cancers by ensuring offspring do not possess the genetic mutation BRCA-1, which increases said cancers risk by eighty and sixty percent respectively.

The power to genetically modify the human genome is being held captive even though it holds the opportunity to cure genetic diseases. The CRISPR method offers “unparalleled potential for modifying the human

Throughout history humans have been able to manipulate various objects and situations in order to further advance society towards an ideal vision. One particular procedure that has gained a significant role towards the importance of the medical society applies to gene therapy and how it has certainly produced an extensive amount of beneficial accomplishments. Gene therapy generally consists of regular genes being switched into a cell in order to replace its defective genes. Introducing a variety of distinct genes into cells of a body can highly raise the risks of them actually remaining functional due to rejection from the body. Due to this process, gene therapy contains a very critical position in the challenges of modern medicine ().

Being able to modify the genes that affect whether or not children have life changing diseases could be a huge step in medical advancement, as well as a large improvement in that individual’s way of life. Ridding a child of incurable diseases before they are even born will save them from a lifetime of hospital visits, hospital bills, sometimes even judgement from others as they are growing up. Although this seems like an ideal solution to a problem very many people have, there are many concerns that could go along with this.

A half dozen cases were found where patients deaths were linked to gene therapy experiments. It was revealed that there were over a thousands of serious unfavorable effects potentially accredited to gene therapy trials, including numerous deaths. In retrospect, researchers have only reported 37 of these intolerable events. Although the hype about gene therapy is keeping legislators in the course of providing “miracle cures”, researchers have defied the most simple ethical beliefs on the use of human subjects. Along with the deficiencies in the technology, resulting in intensifying the regulation of gene therapy trials, most agree that the new policies are far too little, and for the victims of the technology, far too late.While NOT many people actually go through with the medical risks associated with gene therapy, the few that DO experience results, eventually revert back to the disease they started with. Regardless of the countless public protest by some scientists, and lawsuits by health advocacy groups; human gene therapy trials were approved by the federal government with an insufficient negligence and no enforcement. While most of the trials are being unmonitored and practically unregulated

Cross, Deanna and James K. Burmester, Ph.D. Gene Therapy for Cancer Treatment: Past, Present and Future. US National Library of Medicine National Institutes of

Gene is a relatively new but rapidly developing way to treat or cure people of certain diseases. Instead of using a drug or surgery to cure or treat someone they are using gene therapy as a way to insert a gene into the patients cells. If this technique works then it can be an amazing option for someone with a disease with otherwise no cure. Along with this new technique come many risks and dangers that question the ethics involved in doing this. Even though it is only still being tested it can be seen as a promising treatment option for many people.