Cancer and Gene Therapy Gene therapy is an experimental technique that allows doctors to insert a gene into a patient’s cell rather than using drugs or surgery. Gene therapy is a process of which defective or undesired genes in the body with “normal” genes. A vector is re-engineered to deliver the gene to a target cell. Then the gene is transferred to the cell’s nucleus and must be activated in order to function. The main focus of gene therapy is to replace a lost or improper gene with a new functional copy into a vector that is inserted into the subject’s genome by way of penetrating its DNA. Gene therapy can be done outside of the body known as ex vivo by way of taking cells form patients bone marrow or blood and then growing them …show more content…
It has also recently been used in attempt to treat patients with genetic diseases SCID otherwise known as severe combined immunodeficiency, as well as other diseases. Scientist and doctors are optimistic that with time patients can receive a piece of DNA and go home cured. One of the first experiments that was carried out using gene therapy was on a four-year old girl, Ashanti DeSilva. She was born with an autonomic recessive metabolic disorder that manifests as a lack of enzyme adenosine deaminase (ADA). All because of a single defective gene and due to that she suffered from Severe combined immunodeficiency SCID. In the absence of the enzyme, substances toxic to lymphocytes accumulate and immune function is either severely compromised or completely absent. The doctors took blood from their patient and removed her white blood cells. They then inserted the missing gene into the white blood cell, and put them back into her blood stream. The therapy partially restored Ashanti’s immune system. It temporarily stimulated production of the missing enzyme; however it does not generate new cells with functional genes. This strengthened her immune system. Ashanti continued to receive injections of the corrected cells every two months. This helped her lead a normal life.
There are two concepts of gene therapy. The first concept is germinal cell gene therapy which would involve the genetic modification of germ cells.
There are two main groups of gene therapy and they are germ line gene therapy and somatic gene therapy (Baksh, 2007). Germ line therapy consists of germ cells being modified by the introduction of functional genes, which are ordinarily integrated into their genomes (Garbutt et al., 2011). DNA encodes the therapeutic gene and replaces the mutated gene so the new gene can treat the disease. Germ line
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.
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
Gene therapy is the insertion of normal or genetically altered genes into cells to replace defective genes. The gene is usually inserted into
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.
Whether gene therapy should be allowed to be used in practice is currently a very controversial topic. In my opinion, I do not think that it should be allowed. Gene therapy is a technique where genetic material is inserted into a patient to replace missing or defective genes. Gene therapy can be further broken down into two categories: somatic and germline. From there, somatic gene therapy can be approached in two ways: in-vivo and ex-vivo. For the process of gene therapy to begin, the proper vector needs to be acquired. Once a vector is chosen, the vector delivers the new gene into the target cell. Then, the target cell becomes infected with the vector. Next, the vector’s genetic material is inserted into the target cell. Then finally, functional
Gene therapy is an experimental treatment, which is used to replace mutated, improperly functioning genes with regular DNA. There are several gene therapy methods, most of which involve using a vector to transport the corrected gene into targeted cells. Retro viruses, adeno viruses, adeno ascosciated viruses and liposomes are commonly used as vectors. Naked DNA (DNA without
Gene therapy is an experimental medical procedure that uses the genes in our bodies to either treat or prevent disease by introducing new genetic material into human cells (ie. cancer cells) to create the necessary proteins that allows the cell become healthy (SOURCE). Gene therapy can also be used as an alternative to other medical disorders rather than having a surgical procedure done or using drugs such as chemotherapy. For example, Joseph Panno, research
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 could cure many genetic disorders, such as Cystic fibrosis. Cystic fibrosis is a genetic disorder with no cure that causes excess mucus in lungs, digestive tract, liver, and increases susceptibility to infections. In the case that a couple is likely to birth a child with cystic fibrosis, most specialists recommend against the couple having children, as cystic fibrosis can greatly shorten the child’s lifespan. One such case would be a scenario such as this: A couple has
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
Gene therapy is processes of transferring DNA in to a drug as a treatment for certain diseases. Gene therapy was for discovered in the mid 1970’s and it is simply developing a therapeutic form of DNA and putting it into a patient’s cells. In other words this experimental technique in other words uses genes as a treatment or as a prevention to certain diseases. The why this works is that genes are passed down through inheritance. Meaning they go from a parent to its child and then to the child’s child etc. However a certain gene does not produce the right protein or does not produce a protein in a correct manner then a genetic disorder is formed. To dealt with many of these diseases scientist and doctors have been treating the symptoms of the disease but they do cure the actual disease. In other words what they have been mopping and drying the floor from a leak instead of fixing the actual pipe that causes the leak. However gene therapy would be the perfect way for both scientist and doctors to find a way to cure a disease or fix the pipe rather then the symptoms.
Gene therapy could be an approach to alter a genetic issue at its source. By including an amended duplicate of a damaged gene, gene therapy guarantees to help ailing tissues and organs work legitimately. This methodology is not quite the same as conventional medication based methodologies, which may treat side effects yet not the fundamental genetic problems. Most generally, gene