Gene Silencing : Current Approaches And Relevance For Drug Discovery

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.

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

Scientists and doctors alike have long been looking for cures to diseases like cancer. For years now, there have been countless fundraisers to cure this and cure that. And yet, it seems as if there's never any progress. There may be a brief headline in the news about a new discovery, but they always seem to never go anywhere. Maybe the problem is that they've been looking in the wrong places. Maybe the cure doesn't lie on a medicinal level, but instead the genetic level, in genetic engineering and gene therapy. Direct manipulation of an organism's genome can be used in gene therapy as a means to alter genes, causing both positive and negatives effects as well as discussions about the ethical boundaries of modern

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

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 therapy is an experimental technique that uses genes to treat or prevent disease. Currently, there are a lot of studies and experiments that allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or direct surgery. Researchers are testing several approaches to gene therapy, such as replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating, or “knocking out,” a mutated gene that is functioning improperly, introducing a new gene into the body to help fight a disease, and helping to regenerate precious cells. Although gene therapy has a very active and promising approach for a number of disease, there are still several risks and side effects. Hence, the therapy

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

We carry about a thousand genes in us. Most of the genes we inherit come from our parents. Hundreds of genes can contribute to common conditions like breast cancer, Alzheimer’s disease, colon cancer, and rare disorders like cystic fibrosis and muscular dystrophy (Harvard Women’s Health Watch, 1998). Gene therapy is known to treat genetic diseases and other health problems. Figure 1 illustrates how gene therapy works. Genes are introduced into a human’s body by replacing the bad cell with a new cell. In Katherine Hunt’s article, (Gene Therapy), it explains the history of gene therapy that took place between 1970’s through 1990’s. In the 1970’s, scientists came up with “gene surgery,” to treat inherit diseases that are caused by bad genes. In

George Santayana once said, "Those who cannot remember the past are condemned to repeat it," applies equally to technology issues such as gene therapy today. Therefore, I would like to begin by remembering our past mistakes in evaluating Jesse Gelsinger who was 17 when a geneticist told him that his OTC deficiency disorder, ornithine transcarbamylase deficiency, which is an inherited disorder that allows ammonia to accumulate in the blood, could be cured with gene therapy. One day during his treatment he started vomiting uncontrollably and he ended up in the hospital on life support. The vector used to help his immunodeficiency order had deleterious side effects and caused him to have jaundice, multiple organ failures, to be brain dead, and

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

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.

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

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 ().

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.

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.