Gene Therapy And Its Impact On The Medical Society

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.

The gene therapy was discovered in 1972 by authors who are advice the any author to make sure and study the gene carefully, the first try was unsuccessfully, also the first case for transfer gene in the human not count of transplantation of organ, for example it was done by Martin in 1980 , and Cline who is sad that gene was active in his patients after six months, although he did not publish this data, or it has been verified, Even if it is true, it is unlikely that it produced

Genetic disorders have plagued people for ages. However, recent research and new discoveries in gene therapy has brought new hope to those unfortunate individuals that are ailed by their own genetics. Gene therapy is a technique of correcting defective gene sequences specifically responsible for the disease. In 1990, the first successful clinical trial of gene therapy was initiated for adenosine deaminase deficiency (GT). Since then, the number of clinical treatments induced worldwide has increased exponentially. Gene therapies have been a part of modern medicine for almost two decades now. Only recently have scientists started to deliver on their immense promise after a string of setbacks raised doubts about the safety of manipulating human DNA. The idea of gene therapy has been around for a while, but it wasn’t until recently that this field of molecular biology has come more into the light with its advancements over time. Experimentation has been an ongoing process with gene therapy. Although, ethical issues of controversy have surrounded this topic since the beginning. But new facts on gene therapy continues to rebuttal most controversy.

Gene therapy works by adding a good copy of a defective gene to the cells in need (“What is”). Gene therapy is different than traditional treatments because it goes right to the source of the problem, rather than just treating the symptoms (“What is”). Although gene therapy is a possible treatment for many medical conditions, conditions affecting many genes is not the best candidate for gene therapy (“What is”). Inserting the gene into a cell can be an extremely difficult process. To insert the gene into a cell, a virus is attached to a vector (Vachani). This vector is used as a carrier and is inserted into the body with the new gene attached. The gene then goes into the cell’s nucleus and becomes attached (Vachani). There are multiple ways to give gene therapy to people and researchers can find the most effective way. Since gene therapy is still new and being experimented with, the side effects are still unknown (Vachani). After the vector is inserted, the body may react and cause a fever, chills, nausea, and a blood pressure drop (Vachani). These side effects are usually gone after a day or two (Vachani). Although it has not happened yet, researchers are worried that the new genes could start to affect the healthy cells (Vachani). Depending on which cells it affects it could

CRISPR cas9 should continue to advance in the science world because “UC Berkeley researchers have made a major improvement in CRISPR-Cas9 technology that achieves an unprecedented success rate of 60 percent when replacing a short stretch of DNA with another”(Antonio Carusillo, PhD Candidate in Genetic Engineering (Marie Curie) at University of Freiburg (2018-present). This statistic shows that there is more of a chance to success but there is a chance to fail 40 percent but overall it will succeed which is why people are lenient about will it actually work or not, but as technology get better so will treatments to cure hard to pinpoint disease such as cancer, zika, or leukemia. Genome editing (also called gene editing) is a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Several approaches to genome editing have been developed. A recent one is known as CRISPR-Cas9, which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein. The CRISPR-Cas9 system has generated a lot of excitement in the scientific community because it is faster, cheaper, more accurate, and more efficient than other existing genome editing methods (yin, steph.What Is CRISPR/Cas9 and Why Is It Suddenly Everywhere? published april 30,

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

Gene therapy is where normal genes/ genetic material are inserted into the cell where missing or malfunctioning genes are, potentially correcting any disorders caused by the faulty gene. Leading research of gene therapy to treat Cystic Fibrosis has made ground breaking discoveries in ways in which faulty or missing CFTR genes (responsible for moving salt and water out of cells) can be replaced via Vectors, to ensure the protein can regain its normal function. This annotated bibliography provides specifics of key findings and effects gene therapy has in regards to treating Cystic Fibrosis.

We carry about a thousand genes in us. Most of the genes we inherit come from our parents. Hundreds of genes can contribute to common conditions like breast cancer, Alzheimer’s disease, colon cancer, and rare disorders like cystic fibrosis and muscular dystrophy (Harvard Women’s Health Watch, 1998). Gene therapy is known to treat genetic diseases and other health problems. Figure 1 illustrates how gene therapy works. Genes are introduced into a human’s body by replacing the bad cell with a new cell. In Katherine Hunt’s article, (Gene Therapy), it explains the history of gene therapy that took place between 1970’s through 1990’s. In the 1970’s, scientists came up with “gene surgery,” to treat inherit diseases that are caused by bad genes. In

George Santayana once said, "Those who cannot remember the past are condemned to repeat it," applies equally to technology issues such as gene therapy today. Therefore, I would like to begin by remembering our past mistakes in evaluating Jesse Gelsinger who was 17 when a geneticist told him that his OTC deficiency disorder, ornithine transcarbamylase deficiency, which is an inherited disorder that allows ammonia to accumulate in the blood, could be cured with gene therapy. One day during his treatment he started vomiting uncontrollably and he ended up in the hospital on life support. The vector used to help his immunodeficiency order had deleterious side effects and caused him to have jaundice, multiple organ failures, to be brain dead, and

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

A novel therapeutic modality, gene therapy is the transfer of nucleic acids – DNA or RNA – into select somatic (body) cells to correct genetic defects or produce therapeutic proteins. It can be performed ex vivo (“out of the living”) or in vivo (“within the living”). In the ex vivo method, the target cells are removed from the patient, genetically modified, and reintroduced into the patient's body. This approach is efficient but limited to easily accessible cells such as epithelial cells (cells covering internal and external surfaces of the body), muscle cells, blood cells, and stem cells. In the in vivo method, a gene delivery system transfers the genetic material into the target cells within the patient's body. The development of safe

Would you consider altering your DNA if it could save your life? Scientist have been working on gene therapy since the 1970's, this biotechnological form of medicine is the attempt to medically modify cells to help eliminate or prevent diseases by correcting defective genes. Imagine the possibilities of having your DNA tested for heritable diseases and being able to eliminate such diseases from your future.

CRISPR/Cas9 is one of the newest genomic technologies that has completely revolutionized the understanding of disease

I passionately believe that our society depends upon medical advances in order to progress and continue to improve people’s lives. This is achieved by understanding diseases - a disorder of the structure or function that produces specific symptoms or that affects a specified location (McGraw-Hill, 2002) - the effects that they have and the interrelatedness of systems in the human body. Some diseases are genetic – the defective alleles have been inherited from one parent or both. Other diseases that require gene therapy are not inherited and can be caused by a mutation that changes the base sequence in the genetic code. (Mike Boyle, 2008)

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