Human Gene Therapy Essay

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

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

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.

Just in case you are a little shaky on the topic of gene therapy I will briefly explain. Gene therapy is a type of treatment used

Gene therapy is an experimental form of treatment, where normal genes are placed into missing or defective ones in order to correct or alter genetic disorders. In other terms, it is the therapeutic delivery of nucleic acid polymers into a patient 's cells as a drug to treat disease (Gennady, 2015). After many attempts at modifying human DNA by nuclear gene transfer as well as insertion of human DNA in the nuclear genome, the first successful gene therapy was in 1990 where it was used to cure four-year-old Ashanti DaSilva who had Severe Combined Immune Deficiency (IDF 2013 National Conference). Since then gene therapy has been used to cure and treat serious diseases such as Chronic Granulomatus Disorder, Hemophilia, and a wide variety of Cancers, and other genetic and acquired diseases (American Society of GENE & CELL THERAPHY, online).

Gene therapy is a rapidly developing technology first used in 1990.1 It involves the use of modified DNA inserted into an organism with a missing or mutated sequence. There are two methods of delivering this correction into the patient’s cells. In the viral method, a virus containing the vector with the corrected form of the gene, infects the cell naturally. Adenoviruses, adeno-associated viruses, lentiviruses, and retroviruses can all be used. They each utilize a different method of delivery and form of DNA. Adeno-associated viruses and lentiviruses are the easiest forms to construct.2 The corrected DNA faces many obstacles to gene expression and ultimately protein production. The viruses must target and enter the correct cell, avoid

Gene therapy classed in tow types: A)Germ line of gene therapy such as eggs or sperm insert of functional gene, which belong to genomes and the change will be in the new generations. B)Somatic gene therapy, the therapeutic gene there are transferred into the somatic cell of patient and the correction will be in that patient and not including the offspring.Golub, Slonim et al. (1999)

The first method is to attach the gene to a virus. This is done by removing the functional group of the virus that harms the body and attaching the gene that is to be delivered into the cell. Retroviruses and adenoviruses are the two types of viruses used in gene therapy. Retroviral vectors are used because it inserts its own DNA into the host’s DNA. There are two ways to use retroviral vectors in gene therapy, introduce the vector intravenously, or removing the patient’s tissue, inserting the vector in a lab setting, then replacing the tissue. One of the flaws using the retroviral vector method in gene therapy is that integrase enzyme(responsible for integrating the retroviral DNA into the host cell) can insert the gene in any position in the genome. If the gene is place in the middle of another functional sequence, it would cause a malfunction of that particular gene. Not all gene sequences have a significant function, but there is still considerable risk in the retroviral vector method. Although it can be dangerous, this is most commonly used in patients with a small chance of survival, so the risk is not as

Gene therapy is term that offers a wide range of treatment types. These types of

Within this field, somatic gene therapy—the replacement of a faulty gene with a functioning one—is particularly fascinating. One of its interesting aspects is the ability to be customized to the individual. For example, not all patients suffering from a given cancer will have the same mutations. After sequencing a patient’s genome to detect the carcinogenic mutations, doctors can use gene therapy to target and treat the illness at its source. However, at this stage of the technology, gene therapy provides only short-term remission. Studies on blindness showed that the initial success of gene therapy faded over time—an unsurprising outcome since the cells of most tissues will die and be replaced by new, untreated ones. With this area of research still in its infancy, much remains to be done to improve the long-term effectiveness of gene therapy and make it a viable treatment. A potential solution would be to use gene therapy on stem cells as well as on specialized cells. The daughters of the treated stem cells can then differentiate, still carrying the therapeutic gene, and replace the original treated cells as they senesce. For so long, genetic disorders have been seen as something permanent, something scientists could not change. Changing that reality would be immensely

Gene therapy pro-actively transfers new genes into a patient therefore treating or preventing the disease. Gene therapy protocols follow two paths. Somatic gene therapy is a process in which hybrid retro vector is inserted into the cell. The vector binds to the cell membrane, packages itself in the vesicle which breaks down releasing it into the nucleus. Retroviruses are vectors harnessed for this task because their RNA genetic information can easily be transcribed into DNA. Furthermore, retroviruses introduce enzymes like integrase into the cell. Integrase incorporates new genes into the genome of a host. However, integrase can insert the modified genetic material in any arbitrary position in the genome of the host leading to insertional mutagenesis, and possibly cancer (Gene Therapy Retrovirus). Alternatively, cloned DNA solution is physically injected into a solution of zygote pronuclei during germline gene therapy In this way, the zygote produced has modified DNA which is passed to future generations. Nevertheless, germline therapy has a low rate of transgene integration in which modified genes do not get incorporated into the genome of the zygotes (Smith Kevin

How gene therapy is being studied today and what contributions it is making to science

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

Good afternoon Mr David and class. Today the topic of gene therapy will be discussed, so to begin, what is gene therapy? Basically, gene therapy is the modification of genes to alter a certain characteristic that ultimately can cure a genetic disease. There are two types of gene therapy: somatic gene therapy and germline gene therapy. Somatic gene therapy is when blood cells are extracted from a person and a normal gene is then inserted into the defective cell. This does not prevent the disease from occurring in future generations because it doesn’t affect the sperm and egg cells, only the body cells. Although, this is a treatment that has to be repeated several times throughout a person’s life as the effects can wear off. Germline gene therapy however occurs in the reproductive cells where there is genetic modification to these cells occurs. Because these are reproductive cells that are being modified, these changes WILL be passed down to the next generation. Contrasting to somatic gene therapy, germline therapy only has to be done once in a lifetime, as it is permanent.

Gene therapy is the transfer of nucleic acids to a person’s cell to correct a defective gene or cure a disease. The treatment involves actually replacing faulty genes. In the case of hereditary and other lifetime illnesses, it offers a chance of curing a patient permanently of their ailment. After the corrective genes have been replaced in the gene transfer vehicle, the vehicle is injected into the patient, with ex vivo or in vivo injections (Kumar et al).