How Does Gene Therapy Work?

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).

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.

Gene therapy is a way in which an individual with a preexisting genetic disorder (in this case ADA deficiency) can be treated. This involves the insertion of a functioning ADA gene into an individual’s cells through the use of viral vectors, to replace the mutated ADA gene that is non-functioning or functioning incorrectly. In order for this process to be viable multiple techniques are used, including: restriction enzymes, ligation, PCR and transfection. Through these processes an individual is able to

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).

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.

Genes are the functional unit of hereditary and carry the genetic information of the body’s cells. A gene is a DNA sequence that contains instructions for a specific protein. Genes encode instructions that make a number of types of proteins that involved with the growth and development of the human body. Genetic engineering involves the modification of these different traits in organisms. DNA is extracted from one organism and combined with another organism to give new hereditary traits to the organism receiving the traits. Combined DNA is then placed into a host cell and the DNA produces clones of itself. One method of technology used is called recombinant DNA technology. This method creates recombinant DNA molecules that can transform cells into groups of genetically identical cells. Even the slightest changes in combinations can cause significant changes for an organism.

Gene therapy is an experimental technique that uses genes to treat or prevent disease. Currently, there are a lot of studies and experiments that allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or direct surgery. Researchers are testing several approaches to gene therapy, such as replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating, or “knocking out,” a mutated gene that is functioning improperly, introducing a new gene into the body to help fight a disease, and helping to regenerate precious cells. Although gene therapy has a very active and promising approach for a number of disease, there are still several risks and side effects. Hence, the therapy

Human gene therapy is when genetic material is introduced into human cells to treat a genetic disease. Instead of traditionally treating the symptoms, gene therapy aims to treat the disease’s root cause by directly altering genes and correcting defects. Therefore, gene therapy is only viable for certain diseases. Disorders from mutations in single genes are the most treatable, whereas disorders that involve many genes and environmental factors are trickier.

In the last two decades, gene therapy has improved from mental concepts to clinical trials and laboratory experiment. Gene therapy is a technique that is currently being tested to see if genes can be used to treat or prevent disease. This method can successfully be able to replace a mutated gene with a healthy copy, knock out a mutated gene that is not functioning and introduce a new type of gene in the body to help fight a disease a human being may have. Gene therapy is a work in progress system that basically inserts a “normal” genome to replace the gene that is “abnormal” and causes disease.

Misra, S. (2013). Human gene therapy: a brief overview of the genetic revolution. J Assoc Physicians India, 61(2), 127-133.

Gene therapy is a relatively new practice in genetics that aims to correct mutations at a molecular level instead of using drugs or surgical approaches. Different approaches to gene therapy include gene augmentation therapy, gene inhibition therapy, the killing of specific cells, somatic gene therapy, and germ line gene therapy. Through gene augmentation therapy, faulty genes would be taken out of the genome sequence and replaced, effectively ending the effects that the mutated gene had on the body. An example of a gene that would be replaced is the mutated p53 gene. If functioning correctly, the p53 gene should stop the formation of tumors.It works by binding to DNA in the cell and stimulates the production of p21, which when combined with a cell division- stimulating protein, stops cell division. When the p53 is mutated, it fails to stimulate the production of p21 and the cell begins to divide uncontrollably. The gene therapy would replace the faulty p53 gene with a normal p53 gene.

"We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes, "quotes James Watson. This fate that Watson is talking about is contained in our genes, and deals with a new technique, gene therapy. Gene therapy is revolutionizing the world of medicine. Many physicians are predicting that in twenty years gene therapy may change the practice of medicine from a treatment-based to a prevention-based practice. Our future is l ocked away inside of our genes. Gene therapy is unlocking these doors. Researchers are starting to move away from developing new drugs, and towards finding an ultimate solution. That solution is to use gene therapy as a

But many view these risks as subordinate to the potential of gene therapy to help reduce and eliminate the social burden of genetic disorders. The ability of gene therapy to affect such disorders,however, is restricted to those caused by recessive X-linked or autosomal mutations. The realm of efficacy for treatment includes individuals afflicted with common disorders such as haemophilia, color blindness, Duchenne muscular dystrophy, phenylketonuria (PKU), cystic fibrosis, sickle-cell anemia, and Tay-Sachs disease(226-7). The treatment of such disorders must be monitored so that the replacement gene, once inserted, will be expressed at the appropriate time and place, and in the correct quantity(229). Improvements on regulatory techniques are then necessary to enhance the effectiveness of gene therapy. Furthermore, the effectiveness of such techniques is exceeded by the vast majority of the population afflicted with genetic disorders caused by

One of the biggest contributors to the development of the gene therapy approach within medicine was the completion of the Human Genome Project that gave scientists and doctors critical information about the nature of human genetics. It thus became possible to gain a better understanding the connection between certain genes and the development of diseases and disorders linked to them. However, despite the progress that has been made since the completion of the project, there remains a lot still unknown about the human genome and its intricacies. The next generation of medical treatments that will explore this genetic realm will be sophisticated to a level never seen before. The promise and potential that gene therapy has for

revolutionizing medicine and treating disease. It is believed that Gene Therapy holds the key to