Gene Therapy Comes Of Age

Gene therapy has had many successes as well as setbacks. The success of gene therapy outweighs the setbacks Elizabeth would say. The first human to receive gene therapy treatment was a 4 year old girl with severe immune- deficiency disease; the disease was caused by a faulty gene that fails to produce a vital enzyme (Genetics, 23). Gene therapy

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

Li, and Huang discuss the controversial topic of gene therapy by first providing a factual summary of gene therapy. Their introduction goes into the specifics of the types of vectors (carriers that deliver a gene to target cells) used to transfer genes in order to aide damaged proteins. Afterwards, they transition into the detailed of process of gene therapy. They start this off with naked DNA and state it is the simplest approach to nonviral transfers and the least toxic method. However, despite being the least toxic and a nonviral transfer it does not have as great of an effect as Cationic lipids. Cationic lipids have been used in various experiments but are known to be safe only when administered in small doses. Furthermore they go into

The advancing technology allows the reverse of biological aging. In “BioViva Treats First Patient with Gene Therapy to Reverse Aging,” BioViva US is the very first company to try two therapies to try and reverse biological aging. Instead of providing patients with pills, the gene therapy will allow doctors to treat diseases by placing a gene into a person’s cell. In addition, it has been discovered that the main cause of death is cellular aging. Because the aging cell causes Alzheimer disease, memory loss, muscle wasting, and cancer, BioViva is trying to reverse aging and cure the aging cell. Thus, by trying many gene therapies on one person, it might allow them to avoid cellular aging. The CEO, Elizabeth Parrish, even states how this company

In the modern world humans have been able to design and create nearly anything, most to aid us in our daily lives and improve our standard of living. It is only inevitable that eventually humans would take our superior knowledge and skill to manipulate life itself and change our genome to produce a healthier and even more superior human standard of life. In recent years discussion about gene therapy has changed into a promising possibility to treat many of our common human diseases and disorders. Although gene therapy might be the answer to many problems, it has been met with a number of logistical and ethical hardships. With the prospect of being a treatment for inherited genetic disorders, cancers, and viral infections, gene

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.

For many years treatment for cancer has been radiation, chemotherapy, and surgery, but through clinical trials immunotherapy has been tested on various patients that the previously listed treatments did not work for. Immunotherapy is a type of therapy in which the immune system is altered and strengthen to become more rigorous and able to attack cancerous cells. This new discovery is extremely important because it is like a fifth pillar in cancer treatments providing some type of result for doubtful patients.

Researchers have been trying to find new ways to cure disease for centuries. Gene Therapy

Gene therapy is a technique used in attempts to cure or prevent genetic diseases at the molecular level (basically at the source) by correcting what is wrong with defective genes, a good version of the defective gene is introduced into the existing cells. This technique is still considered experimental, only being done through clinical trials. This idea was first suggested during the 1950’s. The basic idea was “if the basses can be arranged incorrectly then why can’t they be rearranged in the correct way?”

Gene therapy is a technique that uses genes as medicine to treat or prevent disease. The technique may be used to replace a defective gene for a healthy gene or use a new gene to favorably change the condition of the faulty gene [1]. There are several problems that challenge gene therapy, the certain condition or disease in question must be well understood, the defective gene must be identified and a working copy of the gene must be available, and the most problematic is effectively delivering working copies of the gene to the cells. The DNA delivery vehicles used in gene therapy are called vectors. There is no omnipotent vector that can treat every disorder. Every gene therapy vector must be customized to address the unique features of the disorder [3].

Gene therapy is an experimental technique that involves altering genes within the body’s cells in order to prevent genetic disorders (Mayo Clinic, 2015). The objective of gene therapy is to edit a defective gene sequence in situ to achieve complete reversion of a disease phenotype for the lifetime of the patient (British Journal of Haematology, 2009). Genes control DNA, the code that controls much of the body’s function and form. Genes that are mutated and deformed can cause disease or disorders. Gene therapy substitutes defective genes or adds a new gene in an attempt to cure a disease or improve the body’s ability to fight against the disease. Gene therapy has the potential to treat or cure a range of diseases and genetic mutations including cancer, cystic fibrosis and haemophilia.

Currently, gene therapy are created and researched to treat diseases that have no cure. Are mostly tested and researched on animals that have similar organs as humans, like mice. Researchers at Baylor College of Medicine have created a high cholesterol mouse and have cured it with gene therapy.

Large amounts of medical treatments are available for combatting and controlling diseases, disorders, and illness. Diseases and illnesses that were fatal to the human body can be cured or prevented through vaccines. However, there are still many other diseases that can’t be cured. Many diseases have drugs and medicine that can help relieve or regulate symptoms of different medical conditions and disorders, but they do not help fix the problem at its source. However, a form of genetic engineering may soon be able to treat these problems. Gene therapy has the potential to be able to treat and cure many diseases and disorders, but it does not come without limitations and

During the 1970’s period, the accepted scientific view of gene therapy was optimistic for future treatments of human diseases, specifically hereditary ones such as sickle-cell anemia or cystic fibrosis.

In collective view, genes are believed as the primary determinant of human health and they’re not modifiable. Thus, genetic disease are still considered as incurable disease. Until this day treatment and management strategies are mostly designed to improve only the particular signs and symptoms associated with those disorders. Although gene therapy become a promising treatment option for genetic diseases, the technique remains risky. Inserting gene into patients’ cells causes an imbalance in the whole living systems. Unwelcome immune response could cause serious illness or even death. Compared to other diseases, genetic diseases affect smaller population. Therefore, from the point of view of pharmaceutical companies, the high cost of developing a treatment makes it an unappealing prospect. A desire to bring a new hope for genetic diseases cure motivate me to pursue a PhD degree in System Biology, one of the programs in Harvard Integrated Life Sciences.