The Importance Of Gene Therapy For Curing Cancer

Genetic engineering allows biologists to manipulate an organism's genomes by utilizing biotechnology (Moulton, 2004 ). The Talimogene Laherparepvec virus, otherwise known as T-vec, is an oncolytic virus. A oncolytic virus, is a virus that destroys cancerous cells. The T-vec virus has recently had successful results for treating melanoma cancer. The virus originated from the herpes simplex virus 1 and has been genetically altered to help destroy cancer cells.The virus gets injected into the lesions, lesions are areas on or in the body that have been damaged . Not only does the virus destroy cancer cells, it also activities the immune system to attack the cancer cells. Many changes were done to diminish the virus, expand the selective for the cancer cells, and release the cytokine. The modifications that were implanted in the virus were, HSV-1 strain (JS1) which is the DNA set of the herpes virus, that increases the death of the tumour cells. To delete ICP34.5, that stops the HSV infection in the cells that are not tumorous, and will provide selective tumour duplication. As Well as the deletion of ICP47 that allows antigen presentation, an antigen is a substance that helps enable an immune response. Placing in the US11 will

So, in one study, scientists engineered a modified CRISPR system to work with customized guide RNA (gRNA) in human cells to target genes linked to leukemia, to which they obtained high targeting rates. They proved that this process was not by random chance, but that it was caused by CRISPR components, that the process was sequence-specific and, when they introduced multiple gRNAs, it can affect multiplex editing of target loci. Their results created an RNA-guided editing tool for easy, efficient, and generalized human genome

CRISPR can be used to treat cancer and other conditions. On October 28, the first clinical trial using CRISPR to treat cancer began in China. In this trial, CRISPR was used to disable the gene PD-1, which cancers use to prevent an immune system response (Cyranoski, 2016). A previous gene-editing method, TALEN, has been used to successfully treat leukemia. A one-year-old girl with leukemia had her immune cells replaced with TALEN-edited cells, which allowed her to survive until a matched T cell donor was found for her (Reardon, 2015). CRISPR is a simpler process and more efficient than TALEN and is predicted to lead to a biomedical race to adopt this new technology between the United States and China (Cyranoski, 2016). Other conditions, such as AIDS, can be treated as well. CRISPR can be used to disable a gene called CCR5, which would prevent someone from developing AIDS regardless of if they are HIV positive. Alternatively, the HIV virus itself could be destroyed using CRISPR (Maxmen,

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

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

Despite the promise of such approaches, a number of difficulties remain to be overcome, the most important of which is the need for more efficient systems of gene delivery. No gene transfer system is 100% efficient, unless germ-line therapy is contemplated. During the past two decades, there have been major advances in our understanding of how cancer develops, proving that cancer has a genetic basis (2). A series of genetic abnormalities that accumulate in one cell may result in a pattern of abnormal clonal proliferation. Our growing understanding of the genetic basis of cancer offers new opportunities for the molecular prevention and treatment of cancer. There has been a

Gene therapy is a relatively new area of medicine that attempts to apply recent advances in molecular biology, genetics and biotechnology to the treatment of human diseases. Gene therapy uses a set of approaches to the treatment of human disease based on the transfer of genetic material (DNA) into an individual. Gene delivery can be achieved either by direct administration of gene-containing viruses or DNA to blood or tissues, or indirectly through the introduction of cells manipulated in the laboratory to harbor foreign DNA. As a sophisticated extension of conventional medical therapy, gene therapy attempts to treat disease in an individual patient by the administration of DNA rather than a drug. (1)

Gene therapy is processes of transferring DNA in to a drug as a treatment for certain diseases. Gene therapy was for discovered in the mid 1970’s and it is simply developing a therapeutic form of DNA and putting it into a patient’s cells. In other words this experimental technique in other words uses genes as a treatment or as a prevention to certain diseases. The why this works is that genes are passed down through inheritance. Meaning they go from a parent to its child and then to the child’s child etc. However a certain gene does not produce the right protein or does not produce a protein in a correct manner then a genetic disorder is formed. To dealt with many of these diseases scientist and doctors have been treating the symptoms of the disease but they do cure the actual disease. In other words what they have been mopping and drying the floor from a leak instead of fixing the actual pipe that causes the leak. However gene therapy would be the perfect way for both scientist and doctors to find a way to cure a disease or fix the pipe rather then the symptoms.

Cancer describes uncontrollable cell growth in a specific part of the body. There are hundreds of types of cancer each with complex biochemistry and cell signalling pathways. The use of CRISPR-Cas9 to detect the initial cause or protein allowing the continuation of the cancer in patients can allow more targeted and personal therapies that could be less harmful and increase the survival rate of patients. The CRISPR-Cas9 mechanism has been used to screen genes that have the potential to be causing cancer by mutating the 5’ end of exons encoding

Modern genetic engineering technologies are advancing, and genetically modifying a genome is becoming a reality. The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated system (CAS9) genome editing system is the most advanced in use. CRISPR can edit a genome more accurately and with higher precision than any other system. The intended use of the CRISPR/CAS9 system is to insert a gene, delete a gene, or silence a gene, leading to powerful applications (Christensen and Choy 2017a). For example, scientists plan to use this technology to change the genome and resulting phenotype in the efforts to eliminate potentially lethal diseases. One such disease is Human Immunodeficiency Virus (HIV) and Acquired

Gene therapy is a process involving the transplant of non-mutant, normal genes into cells, either in place of missing genes, or to essentially replace those genes which are not functioning properly, or not coding for properly functioning proteins, with the purpose of treatment and correction of a number of genetic disorders. This particular type of treatment technique is hoped to, in the future, be able to replace surgery, and a slew of different drugs in the treatment of a variety of diseases in humans caused by genetic defects. This seems to be a promising technique with the potential to redefine the way we approach treatment of many crippling diseases causing human suffering, however, the development of such a technique raises some questions. For one, what exactly is the mechanism of action utilized in such a technique, how is the functional gene translocated to the target cells, and once there, how is the new, functional gene integrated into the target cells in order to effectively “treat” the disorder. Another question is, what clinical diseases and disorders would this treatment be appropriate for, in addition, which disorders have been attempted to be treated with this technique already, and have there been any successes with this treatment technique. Finally, have any ill effects, short or long term, been observed in patients treated with this gene therapy technique? If so, what are they, are these effects irreversible, and have there been any studies into the

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

The world of genetics has mainstreamed into medical practice, transforming the way patients are treated. Recently, gene therapy has been propelled into the spotlight with the most recent successful clinical trial in which scientists reprogrammed ordinary cardiac cells in pigs to mirror the function of the sinoatrial node, thereby inducing a steady heartbeat. In the last few decades since its inception, gene therapy has rapidly advanced; there have been remarkable strides in its methods and an increase in the range of targetable diseases.

To what extent are retroviral, adenoviral, and adeno-associated viral vectors effective in transferring and integrating therapeutic genes?

Genome editing generally had a large time-scale and heavy cost associated with it, however, that is no longer the case due to a genome editing tool named CRISPR-Cas9. This tool is based on a natural system used by bacteria to protect themselves against viruses in which the bacteria disables the virus by cutting the viral DNA with an enzyme called Cas9. The adaptation of this naturally occurring phenomenon into CRISPR-Cas9 has opened the door to many incredible possibilities that were previously thought of as impossible. One possible use of CRISPR-Cas9 may be the curing of retroviruses. In 2015, scientists used CRISPR-Cas9 in the treatment of rats with HIV. The persistent co-expression of the Cas9 enzyme and specific guide RNA significantly diminished HIV-1 replication in infected CD4+ T cell cultures, which saw more than 50% of the HIV-1 virus being eradicated from the rats, as well as them being protected against new infection (Rafal K et al, 2016). This may serve as a pathway to the curing of HIV and other retroviruses. CRISPR-Cas9 could also be used to tackle one of the largest causes of death today, cancer. Cancer is caused by the replication of altered cells that are not recognised by the immune system, causing tumours. CRISPR-Cas9 has already been used to treat cancer in mice. Using Crispr, scientists modified the immune cells of the mice to express proteins on their surfaces called Chimeric