Stem Cell Gene Therapy Essay

Originally in Takahashi and Yamanaka’s experiment, retroviruses were used as a vector to introduce factors that triggers reprogramming, however with evidences from further researches shown that retroviruses may cause insertional mutagenesis, where the host’s DNA is mutated due to the virus integrating into the host’s genome, and potentially cancerous nature of the iPSCs made this technique ill suited to further iPSC developments.(5) Therefore, other methods have been developed and used nowadays to minimise these issues and they include protein transduction, the use of

This article covers the Seneca Valley Virus (SVV-001) as a hopeful for an oncolytic treatment of certain cancer types. More specifically those with neuroendocrine properties such as rhabdomyosarcoma, Wilms tumor, glioblastoma, neuroblastoma, and adult small-cell lung cancer. Each of which effect smooth/skeletal muscle cells, kidneys/adrenal glands (mainly in children), astrocytes of the brain, nerve cells of a fetus, and lung cells in adults respectively. The virus was discovered by accident in a contaminated cell culture that contained bovine serum to promote growth. The virus was later discovered to be almost exclusively found in farm animals such as cows and pigs, due to the presence of neutralizing antibodies that were later to only ever have been found in one human sample. Just as important as that, the virus only targets the cells of the above-mentioned cancers/tumors, is a self-replicating RNA virus, and its inability to infect other cells in the body all come together to result in the lysis of these specific cancer cells. These properties alone give great hope for SVV-001 as a treatment for those infected by these diseases, and prompted for more research into its medicinal possibilities.

Stem cell research is the future of medical and biological research and remedies, and it is fascinating to watch the progression of this new and important science as it unfolds. These cells were discovered in mouse embryos in the 1980s, and are remarkable because of their potential to grow into a variety of different kinds of cells within a body. Common in fetuses, and more rare in adult animals of all kinds, stem cells can be manipulated in useful ways to repair many tissues, dividing limitlessly for therapeutic purposes. When a stem cell divides, each new cell has the potential either to remain a stem cell or to differentiate into more specialized tissue, such as nerve, pancreas, bone marrow, or unique blood components. Initially

The use of stem cells can advance drug development, knowledge of disease, patience specific disease treatment, and can bypass the limits of mouse-models for research. According to the National Institute of Health (1), stem cells are unique in the following ways: they can divide and renew themselves for long periods, they are unspecialized, and they can give rise to specialized cell types. The uniqueness of these stem cells allows for the testing of new drugs, cell-based therapies and the study of human development including cancer research. Human stem cell therapies have been used for the treatment of neurological disease in human clinical trials such as Parkinson’s disease (2), spinal cord injury (3,4) stroke (5), and

Gene therapy is a relatively new method for treatment of chronic illnesses that are hard to treat with other methods. So far, it has been considered as a possible treatment in conditions such as cancer, cystic fibrosis, AIDS, and ALS. Recently, research has been done on using gene therapy as a treatment for the symptoms of neurological and nervous system disorders, which includes Parkinson’s disease and Alzheimer’s. As with any new treatment, the main question to consider when thinking

Virus-induced neoplasia may also be subjected to additional host and environmental factors that may modify the transformation process. Studies have shown that there are Cellular genes that are homologous to the transforming genes of the retroviruses, a family of RNA viruses that can induce oncogenic transformation

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

Reading an article and learning about “tumor-busting viruses” is very compelling, to say the least. Since it seems almost everyone has endured the pain of cancer or has a loved one who has been diagnosed with some form of cancer, it is refreshing to see that researchers and scientists are coming together to find fresh techniques to help heal this tragic illness.

The society has evolved over the decades. Medical advances, greater technologies, better understanding of the world around us, the sky is the limit! The progression of a society is astounding from where it was just twenty years ago. One is able to do things that the forefathers would have never dreamed! Science has discovered ways to travel to the moon and back, reach some of the deepest depths of the oceans and discover new species, and drive hybrid cars. The achievements of today’s society have reached a new high; however, with great achievement comes great questions of ethics.

Without a vector, mutated cells cannot be targeted, which means the treatment would be ineffective. Since the 1990’s, scientists have experimented with the use of viral vectors, such retroviruses, adeno viruses and adeno asscociated viruses. Viruses contain a circular piece of DNA known as the plasmid. During gene therapy, the segment of DNA responsible for viral spread is removed. The correct copy of the mutated gene is then inserted into the virus. The virus is then inhaled,swallowed or injected depending on the location of the target cells. (in the case of CF, the vector is inhaled). Retroviruses are viruses that contains RNA instead of DNA. Retroviruses can only infect dividing cells and the viral RNA needs to be reverse transcribed to DNA, so that gene can be replicated for mitosis (this is done using enzymes). Unfortunately, retroviruses have been found to implant genes into random parts of the genome. This means that the introduced gene can disrupt an existing, functional gene. Between 1999 and 2006, reasearchers performed gene therapy on 5 children with a genetic immune defficiency, using retroviruses as vectors. By 2006, all 5 of these children had developed leukemia as a result of the vector implanting into a random part of the genome. Adenoviruses have also been used as vectors, however, they do not integrate into the genome permaneatly and are likely to trigger an immune response. In 1999,

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

In 1990 the first gene therapy procedure gained approval and a four-year-old girl with SCIDs disease was finally able to fight off a simple cold. She is now able to live a normal life with the help of continued treatment of gene therapy. Although gene therapy is an innovative and ideally favorable procedure when it comes to treating diseases, cancer, or inherited disorders, it is still a delicate procedure and is continuously studied to insure it is not only effective but also and most importantly safe. In order to insure the safety of patients a gene therapy drug must get the approval of the FDA before becoming available, as every drug must do. Since gene therapy is still very experimental; there are only a few gene therapy drugs that have been approved by the FDA to treat patients. However, on August 30th, 2017 history was made when the FDA approved Kymriah, the first CAR-T cell therapy drug to be available in the United States. Kymriah is a one-time treatment for patients with B-cell acute lymphoblastic leukemia, it was developed through the research collaboration of Novartis and the University of Pennsylvania, and it is manufactured for each individual patient. Kymriah’s only drawback would be the price tag of $475,000 for a course of treatment, however Novartis plans to wave the fee for patients where Kymriah is not successful.

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

There are three techniques that researchers are working on. The first and most common is ex vivo ( or "outside the living body") therapy. The defective cells are removed from the patient and replaced with the normal DNA before returning to the body. This therapy targets the blood cells because many genetic defects alter the functioning of one type of these cells or another. But since blood cells have limited life spans follow-up treatments are required. Future efforts will most likely target stem cells of the bone marrow. Stem cells are ideal for gene therapy because they appear to be immortal. Researchers have obtained stem cells from human bone marrow, but they are having difficulties getting genes into the cell as well as inducing the cells to produce many new blood cells (Anderson, 1995).

The DF-1 cell line is often used in the research of avian viruses. It is unique to the group of mammalian cell lines in that it does not contain endogenous sequences linked to avian sarcoma and leukosis, a first of its kind in continuous avian cell lines. The study of avian retrovirology relies heavily on access to a continuous cell line, and the DF-1 cell line is both continuous and non-transformed. Because the cell line lacks any sequences related to avian viruses, it is most popularly used to study the effects of these viruses on cells as they