Double-Edged Sword

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.

Roughly fifty years ago, scientists thought that the use of genetic modification by exogenous DNA would be effective. This idea could offer curative and long lasting benefits, in a single treatment.

There are two types of gene therapy; germline gene and somatic gene therapy. Germline gene therapy is considered to be a safer option for humans. This method makes changes to the gamete cells that are used in the reproductive process thus, the functional genes need to be inserted into the chromosomes. Somatic gene therapy is when genes are transferred to body cells by inserting vectors into a person’s body. There are three things that gene therapy can do; replace a mutated gene that causes a disease with a healthy gene, deactivate a mutated gene that is not able to function properly, or introduce a new gene into the body to prevent diseases. There has been a clinical trial in the past where over 3000 people were treated with gene therapy and the results were good however, this treatment method has serious health

Gene therapy is a relatively new technology that has shown some promising results. Such as the drug called Strimvelis that is used to help children with severe combined immunodeficiency, a disease that leaves infants with little to no protection against viruses, bacteria, fungi, and other potentially harmful things. It appears to be a cure carried out through a genetic pair. Strimvelis was tested on 18 children, starting 15 years ago, all of whom are still alive today. Gene therapy is used to inject genes into a person’s cells and tissues in order to treat a disease, as well as hereditary diseases where an abnormal allele is replaced with a fully functional allele. The carrier, called a vector, is used to transport the normal gene to the target cells. The most common type of vector are viruses that have been genetically altered to carry human DNA. Scientists have been trying to control the way the viruses have evolved encapsulating and delivering their genes to normal cells in a pathogenic way by manipulating the viral genome to remove disease-causing genes and inserting healthy ones. Theoretically, it is possible to transform either the cells of the body (somatic) or the cells of the sperm, ova, and their precursors (germline). All of the gene therapy experiments so far on humans have been directed to somatic cells, because germline experiments have been remaining controversial. Somatic gene therapy is split into two broad categories: ex vivo (cells are modified outside of the body then returned back again) or in vivo (genes are changed in cells while still located inside of the

Gene therapy has the potential to revolutionize modern medicine. The techniques of gene therapy are still in their infancy as medical treatments and there are still many problems which must be solved before gene therapy will live up to its potential. However, it is very likely that gene therapy will become a reality at some point in the future and when that time comes, the ethical questions surrounding gene therapy will be pushed to the forefront of medicine. Science may find a way to reduce the risks associated with gene therapy but science alone cannot eliminate the serious ethical and societal risks which gene therapy brings to bear on the world. We need to put people's

A new form of medicine with the potential to save and improve millions of live is gene therapy, or gene replacement therapy. Gene therapy is the use of DNA as a pharmaceutical agent used to treat or cure diseases or disorders caused by defective or missing genes. There are three methods of gene therapy: replacing a mutated gene with a healthy copy of the same gene, inactivating a gene that is not functioning properly, or introducing a completely new gene to help the patient fight a disease. Gene therapy is a promising treatment option, however, the technique has yet to be perfected and is still dangerous and experimental.

Gene silencing, the ability to selectively suppress the expression of a single gene, is something that was once thought of as a “holy grail” in medical technology. The potential treatments for this technology include, but are not limited to, inhibiting viral infections, cancer replication, and certain genetic disorders. Considering the potency of each of these problems within the modern medical field, the potential economic and physiological impact of gene silencing is massive (amounting to billions of dollars in investment). With the recent discovery of RNA interference (RNAi) (Fire and Mello) and the field’s development within the last 20 years, the impossible is starting to become possible.

As years go by and technology advances, many medical techniques that failed in the past are starting to seem more possible. Researchers are discovering that a technique called gene therapy is able to help prevent and reverse the effect of many conditions. Originally in the early 1900's, gene therapy failed in

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

According to Blau and Springer (1995), gene therapy is a newly rising approach at the end of the 20th century and aims to open new door for the treatment of various human diseases based on transfer of genetic materials to cells. The recent gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy is not inherited and thus it cannot be passed down on to a person’s children in the future. Meanwhile, gene therapy could also be targeted to egg and sperm cells (knowns as germline gene therapy) which would allow the inserted gene to be passed on to the progeny. Germline gene therapy allows the correction of disease-causing

Since 1990, gene therapy had been tested in human clinical trials for treating such diseases as severe combined immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease, and Gaucher's disease. ("Gene Therapy," n.d.). In the United States, gene therapy is only available in a research setting.

The process of gene therapy is the introduction of a “good,” or normal, gene into a person who is affected by a “bad,” or mutated, gene. The doctor is manipulating the genes in the child to try and treat whatever disease the individual has. There are 2 types of gene therapy, somatic and germline, and in both ways there is no way to get away from the disease. In somatic gene therapy, the doctor injects the normal gene into the patient’s targeted cells to try and treat them, but the new gene does not get passed on to the offspring of the patient. In germline gene

There are many organisms that use RNA (ribonucleic acid) interference, also known as RNAi to control genes, scientists can also use RNA interference as a tool in the laboratory and in the future may be able to use it as a therapy. RNA interference is described as the biological process in which RNA molecules prevent gene expression. Eukaryotic cells have many ways of controlling gene expression, as in the complex environment in a cell these mechanisms need to be precisely targeted. Small RNA molecules are used to direct gene silencing (or gene knockdown), this is called RNAi. Gene knockdown can be described as the technique used to reduce the organisms genes, this can occur through either genetic modification or treatment with a reagent

Many scientists and others believe that if gene therapy can be refined, it could be implemented to ultimately put an end to genetic disorders. The parents would have the children they want, and the children who had the natural disposition towards a genetic disorder could possibly become a normal child, whereas otherwise they would probably end up losing their life due to abortion. Those that support gene therapy view it as a win-win strategy:

Every living thing is the product of the genes that were passed down from ancestors. Genes make up everything we are. One gets their traits from their parents. Most people live full lives with relatively good health. However, some people inherit mutated genes or faulty genes. This could lead to genetic disorders that could be life threatening. Even today, many genetic disorders still remain incurable, leaving many people without hope. Genetic therapy could be their answer. It is through this research that the cure for genetic disorders can be found. Though some people believe it is unethical or immoral to alter genes, current therapeutics have not been able to save the lives of the patients with these diseases. Genetic therapy