Implications of and Attitudes toward Gene Therapy

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

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 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 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

With our current society rapidly advancing in both the technological and medicinal world, many new treatments have been born that can be used to treat genetic conditions that regular antibiotics and surgeries simply have no positive or progressive effect. Gene therapy is when a newly developed gene is transferred, or introduced, to an already existing gene, it is the attempt to treat one’s genetic disorder at the molecular level and could significantly improve the human population and provides hope to people with disabilities. Gene therapy goes straight to the source and alleviates symptoms of the disorder. The gene receiving this new development is in some shape or form, mutated, or defective. If the gene therapy is a

Gene Therapy is an experimental form of treatment that aims to get rid of genetic conditions at their source. It also may be used to replace a faulty gene with a working version or introduce a new gene to cure a condition or modify its effects. ( Victoria State Government, 2011) It is designed to introduce genetic material into cells to compensate for abnormal genes or to create a beneficial protein within the gene. If a mutated or incomplete gene creates a protein needed that is faulty or not present, gene therapy has the ability to introduce a copy of that specific gene to restore the function of the protein within that gene.

The objective of gene replacement therapy is to alter the defective gene to achieve a permanent cure for the diagnosed patient by identifying the faulty gene and providing a corrected copy of the gene without affecting the genomic sequence. Clinical trials initiated gene replacement experimentation, primarily leading to significant progress but not yet accomplished.“The most recent clinical trial for the treatment of hemophilia with gene transfer showed transient achievement of efficacy in the highest dose cohort tested, but also exposed a previously hidden barrier to the future success of these treatments.” (Gene Therapy for Haemophilia) Recently, the focus of gene therapy strategies is mostly on gene addition rather than gene replacement.

Since its inception, gene therapy has captured the attention of the public and ethics disciplines as a therapeutic application of human genetic engineering. The latter, in particular, has lead to concerns about germline modification and questions about the distinction between therapy and enhancement. The development of the gene therapy field and its progress to the clinic has not been without controversy.

Such genetically modified viruses are called vectors, and are the most popular way in which to administer gene therapy. Additionally there are many ways that these vectors could be introduced to potential patients of gene therapy. According to Genetics Home Reference, a government authority for genetic information, “a vector can be injected or given intravenously directly into a specific tissue in the body… Alternately, a sample of the patient’s cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient.” (GHR 2) However getting corrective DNA into a host is only half of the challenge.

Gene therapy is the introduction of nucleic acid, including DNA, RNA and their analogue into cells of living organism to treat diseases. This is occurred through modifying expression of gene of interests, triggering alteration of certain biological functions.

Gene therapy is a technique that is used to cure diseases, and to stop malfunctioning genes from producing the disease in humans. Gene therapy involves introducing normal functioning genes into a sequence containing malfunctioning genes, which either stop or hinder the production of important proteins or cells needed in the body. Doctors are able to cure diseases, and may soon be able to prevent diseases, by taking copies of the DNA sequence with the malfunctioning gene, and inserting a better working gene into the DNA sequence, which will then provide the cells with the correct amount of proteins that it needs to carry out bodily processes. The most common techniques of gene therapy used in order to cure severe combined immunodeficiency diseases are, Restriction enzymes, PCR, DNA sequencing and Gel electrophoresis, Ligation and viral vectors. These four techniques allow scientists to insert functioning normal genes into the area causing the disease, and produce the correct amount of proteins needed to cure humans.

For instance, a technique known as gene therapy is the transfer of genes into a patient’s cell as a drug to treat or prevent a disease. Gene therapy can include replacing a mutated gene, removing a gene or the addition of a new gene (Hulot et al, 2016).