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 is the process of replacing a defective gene inside a patient¡¯s DNA with a working gene that will produce the correct gene products. The genetic diseases ¡°in which a single known gene does not function properly¡±, such as sickle cell anaemia, thalassaemia and Lesch-Nyhan syndrome, are most suitable to be treated with the gene therapy. There are two types of gene therapy in curing these diseases, patient therapy and embryo therapy. In the process of the patient therapy, the first step is identifying the defective gene and isolating a normal counterpart. ¡° To obtain correct gene action, it may be necessary to put it into the correct site on the host cell chromosome, or even to delete the defective gene¡±, and the DNA can then be replicated each time the host cell divided.
Viruses (that have been modified to not cause harm) are well known vectors for gene therapy. Retroviruses and adenoviruses are commonly used viruses that are used for gene therapy; they may or may not incorporate their own genes into the gene being delivered to the human chromosome. The vector could be injected by an IV into a specific part of the body in which, it can be taken up by individual cell (invivo). Another option could be to take a cell out of the person’s body and in a laboratory place the vector in the cell (exvivo). Later the ce... ... middle of paper ... ...efore we know it doctors will be prescribing genes instead of pills or painful injections.
Gene therapy is the insertion of normal or genetically altered genes into cells usually to replace defective or diseased genes in the body. By using gene therapy, doctors can go to the source of the disorder or problem instead of prescribing a patient a countless number of drugs (Hogarth 1). Gene therapy was designed to introduce new and corrected genes to compensate for any abnormal genes. If a mutated gene causes a protein to be missing or to fail, gene therapy is a way to inject a new and normal copy of the correct gene to revitalize the function of the lost protein (Genetics Home Reference 1). With the help of medical advances in technology, gene therapy has gone from the idea stage, to technology development and laboratory research, to clinical trials for various disorders (ASGCT 2).
Another molecule of DNA that had also been snipped with the same restriction enzyme was found to have a corresponding sticky end that could combine with the original sticky end to form what scientists call a recombinant DNA molecule. By using restriction enzymes, scientists can cut genes out of chromosomes in order to reinsert them into other ... ... middle of paper ... ...eld of medicine. Both genetic engineering and stem cell technology are essential branches of biotechnology. Genetic engineering is the skillful manipulation of a gene by way of a process other than ordinary reproduction. Stem cells that won’t be denied by immune systems, can divide almost forever, and can alter themselves easily into other cells are used in stem cell technology and hold much promise in the cure of diseases and injuries.
According to Yourgenome.org, Gene therapy is basically when the DNA is introduced into a patient to treat a genetic disease. Precisely, the new DNA contains a functioning gene typically to correct effects of a disease-causing mutation. Typically, there are two types of gene therapy, but they are different depending on the kinds of cells treated. There is somatic gene therapy and germline gene therapy. Additionally, there are also some techniques used to carry out gene therapy.
Gene Therapy Gene Therapy is the insertion of a gene or genes into cells in order to provide a new set of instructions to those cells. Gene insertion can be used to correct an inherited genetic defect which is causing disease, to counter or correct the effects of a genetic mutation, or even to program a cell for an entirely new function or property. Genes are composed of molecules of deoxyribonucleic acid, or DNA (see Nucleic Acids), and are usually located in the nucleus of cells. The instructions that direct the development of an organism are encoded within the genes. Some diseases such as cystic fibrosis are caused by an inherited genetic defect.
For example, hemophilia is caused by the mutation of a single gene. The most obvious targets for gene therapy are single gene defects, where a single, faulty gene causes a disease. While easy, single gene targets for gene therapy include hemophilia, cystic fibrosis, muscular dystrophy and SCID, gene therapy may have the potential to cure HIV, malignant melanoma, kidney cancer, Gaucher disease, breast cancer and lung cancer (Sinnott). Since the 1970’s, scientists have been manipulating genes through gene splicing. Gene splicing involves removing a fragment of DNA containing the specific DNA sequenc... ... middle of paper ... ...cientist will have to learn how to turn on specific genes when a protein of enzyme is low, and off to avoid too much to ensure the delicate chemical makeup of our bodies is not disrupted.
By natural breeding genes are passed down to offspring by the parents however, with new technology scientists are able to identify an individual gene and insert it into another organism which will then carry the trait of that gene. This alters the DNA. The extraction of the gene is done by physical and chemical methods The process of moving a gene from one molecule to another molecule is called cloning. In the cloning process a fragment of DNA, containing a gene is cloned into a vector where it is grown in large quantities and manipulated in a variety of ways. The most common vectors are bacterial plasmids but viruses and self-replicating units in eukaryotic cells are also employed as vectors.
Mechanism of Transfer in Gene Therapy Abstract: Gene therapy is the transfer of “normal” genes into the body to replace defective or undesired genes. The transfer may be in somatic or germline cells and may take place in vivo or in vitro. The DNA may be inserted in a retrovirus, adenovirus, adeno-associated virus, herpes simplex virus, or liposome, or it may be naked DNA. The vector travels to a target cell and inserts the gene, which goes to the host cell’s nucleus and may integrate into the genome. In cells with dominant negative genes, resulting protein must be prevented from translation or transcription using oligonucleotides or antisense technology.