The Human Gene Therapy

Gene therapy is a very controversial topic that has been discussed in the last five years and is being heavily studied to help cure cancer. Gene therapy is a technique aimed at treating genetic disorders by introducing the correct form of the defective gene into the patient’s genome (Dunlop et al., 2010).

DNA has about 12 million single nucleotide polymorphisms and thousands of copy number variants, most of which are not harmful. Sometimes these mutations in genes can cause genetic disorders that end up altering or inhibiting protein function. Gene Therapy focuses on correcting the mutated genes usually by inserting a normal gene into the genome. Researchers treat these diseases this way by repairing dysfunctional gene or by providing copies of the missing gene. They isolate the normal DNA and package it into a vector. Other ways include replacing the abnormal gene with a normal one, repair the mutated gene, or altering the regulation of a certain gene. As of right now, reparative results have been marginal, there is potential of gene therapy treating single-gene disorders.

Gene therapy is an experimental technique that is used to prevent disease. For future reference, this technique may treat disorders. It may add or remove genes to get rid of a genetic disorder. Gene therapy is a heavily debated subject. There are very respectful points on both sides of this debated topic. The pro side believes that gene therapy is the best thing that is happening to our society. However, opposers of gene therapy believe that people should refrain from proceeding with this biological advancement and it is ethnically incorrect.

Gene therapy: Is a treatment method that involves the manipulation of an individual's genetic makeup. A form of therapy that attempts to fix the defective gene which is causing the disease.

Gene therapy is an experimental technique that uses genes to treat or prevent disease. It is currently only being tested for the treatment of diseases that have no other cures. There are three different methods of gene therapy: Replacing a mutated gene with a healthy copy, inactivating a mutated gene and introducing a new gene in the body to help fight the disease. This can only be used on certain diseases and is very risky. (Genetics Home Reference) Usually gene therapy uses a vector, typically a virus, to get the gene to the correct cell. When it is inside, the cell's gene-reading machinery uses the information in the gene to build RNA and protein molecules. The proteins can then carry out their job in the cells. (What is Gene Therapy?) Gene therapy is not available for familial dysautonomia at this moment. Even if gene therapy were an option I would not want to go through with the procedure because it is very risky and has a very high chance of you getting even sicker and a very low chance

Some people may wonder what gene therapy is. Gene therapy is the replacing of defective or missing genes with normal genes in order to cure the disorder. In the future, this technique may be used to prevent or treat a disorder by inserting

Demolishing genetic diseases from the human race has been and always will be a key role in science. However, there comes a point in time when moral standards have interjected the betterment of cleansing genetic diseases. One of the many branches of science that has the complete capability to exterminate our destiny of any genetic diseases, gene therapy, is being silenced due to the curse of society’s moral standards. There is a very narrow line in what is not morally correct and ethical to standards of people; causing the advancement of procedures like gene therapy to be narrow. The only explanation to gene therapy awaits on what we, as a community, can bring about for the future generations, but this is

Fifty years after the idea of gene therapy was first proposed, gene therapy has become a possible treatment for a couple different diseases. Before this treatment was approved, some serious unfavorable effects were found in clinical trials. However, these effects fueled more basic research in order to improve, in efficiency and safety. Gene therapy has been used for patients with blindness, neuromuscular disease, hemophilia, immunodeficiencies, and cancer.

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

Li, and Huang discuss the controversial topic of gene therapy by first providing a factual summary of gene therapy. Their introduction goes into the specifics of the types of vectors (carriers that deliver a gene to target cells) used to transfer genes in order to aide damaged proteins. Afterwards, they transition into the detailed of process of gene therapy. They start this off with naked DNA and state it is the simplest approach to nonviral transfers and the least toxic method. However, despite being the least toxic and a nonviral transfer it does not have as great of an effect as Cationic lipids. Cationic lipids have been used in various experiments but are known to be safe only when administered in small doses. Furthermore they go into

Deoxyribonucleic acid (DNA) was discovered in 1944 by Avery and colleagues. Avery identified DNA as the primary genetic material. Watson and Crick later discovered the double helix structure of DNA. Leder and co-workers deciphered the triple nucleotide code that designated the amino acids from which proteins were built. The science of molecular biology was born (Sokol, Gewirtz, 1996). In 1990 a four year old girl who was suffering from severe combined immunodeficiency (SCID) was the first to undergo gene therapy. White blood cells were removed from the girl and the cells were inserted with normal copies of the defective gene and returned into the girls circulation. Her condition improved with four treatments and

“Although we are all members of a single species, we differ from one another in such visible traits as the color of our skin and the shape of our noses, and in biochemical factors such as our blood types and our susceptibility to certain diseases” (Sheridan College Institute of Technology and Advanced Learning, 2013, p. 34). To understand human physical development and evolution one has to understand biological anthropology as the focus on humans as biological organisms. Biological anthropologist conduct research, and form techniques of modern molecular biology to learn about human variation and how it relates to different environment humans lived in as well as their conditions.

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:

The word science comes from the Latin word "to know" (Science Made Simple, 2014). In

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