Objective: Gene Therapy is one of the upcoming targeted treatment options. An attempt to understand the methodology for this therapy, look at the progress made/success stories, road blocks and future potential of this therapy option for patients. Background: In 1990, the first human gene therapy experiment was conducted on four year-old Ashanti Desilva who suffered from adenosine deaminase (ADA) deficiency. Though a complete cure was not obtained it did correct the deficiency with reduction in expensive treatment costs. In 1999, 18 year-old Jesse Gelsinger suffering from OTC disease (ornithine transcarboxylase deficiency), was the first reported death to be directly caused by gene therapy treatment. 1 Gene therapy has come a long way with …show more content…
In Somatic gene therapy inserted genetic material to the target cells, does not pass on the change to the next generation. In comparison the opposite is true for Germ line gene therapy. Due to this evident difference, current regulations allows gene therapy only on somatic cells.1 There are two lines of treatment applications for gene therapy, one for the treatment of genetic diseases and the other for non-genetic and polygenic disorders.3 Two different approaches to administer gene therapy to patients exist. “In vivo” which involves a therapeutic gene being administered directly via a vector into the tissue of the patient. This vector interacts with the target protein to produce the required therapeutic protein. The second approach is the “ex vivo” gene therapy, specific cells are isolated and purified from a patient, the vector is introduced following which these genetically modified cells are re-infused. Vectors in Gene Therapy The Vector, hence is a key delivery medium for gene therapy. A vector can be described as a system fulfilling several functions, …show more content…
Viruses are therefore employed as a medium to deliver the required gene with high efficiency into the target cells. Viral vectors are engineered using sophisticated methods such that the unwanted genes responsible for replication, assembling, or infection are replaced by the therapeutic gene. Current Viral vectors can further be categorized into, Non-lytic viruses, retroviruses and lentiviruses, produce virions from the cellular membrane of an infected cell, leaving the host cell relatively intact and Lytic viruses, human adenovirus and herpes simplex virus families, destroy the infected cell after replication and virion
In 1986, William French Anderson and his team discovered and perfected gene therapy for ADA-SCID, correcting T-cells outside the human body and safely administering them back into the body. Three years later, DeSilva, barely four years old, successfully received gene therapy, and DeSilva’s immune system became fully functional. SCID gene therapy helped pave the way for other treatments to
Instructions: Answer the questions as directed. Upload the assignment prior to the beginning of your next lab section. Make sure you give yourself time to troubleshoot any issues you may have with uploading the assignment. You are responsible for uploading the correct file. Given the map of the plasmid in Figure 10-3, you should be able to predict the length of DNA fragments that will result when these digests are completed.
During the 1970s it became possible to introduce exogenous DNA constructs into higher eukaryotic cells in vitro. Mammalian (germline) trans genesis was first achieved in the early 1980s. The model used in this study was mice. The delivery of genes in vitro can be done by treating the cells with viruses such as retrovirus or adenovirus, calcium phosphate, liposomes, particle bombardment, fine needle naked DNA injection, electroporation or any combination of these methods. These are the powerful tools for research and have possible applications in gene therapy.
Just as with germ-line gene therapy, there are risks with implementing any developing medical, surgical or pharmaceutical treatment. However, it is argued that the advantages outweigh any negative consequences. If germ-line gene therapy is performed well, there is the potential to eliminate diseases that, as of now, are considered irremediable. An example of such idea observed in the present is the administering of chemotherapy to treat different kinds of cancer.
The human body has 20,000-25,000 individual genes and the question has arisen of who owns those genes. While having a patent for part of the human genome is different than owning those genes, it has some of the same implications. Thus, the question arises should human genes be patented, and what are the various implications of patenting genes and DNA? Finding a solution to whether the human genome should be patented can be difficult because it requires balancing moral, social, technological, academic, and economic concerns. Even more than that, these concerns need to be addressed in several areas such as naturally occurring DNA, non-natural or lab-synthesized DNA, as well as treatments based on the patients’ genetics.
If pharmaceuticals and scientists pair and discover new material, gene editing could bring even more money to the table. With advanced research comes advanced medicines and treatments. Potentially, with gene editing we could become a healthier, more advanced way of
Eugenics: Addressing the Line Between Utopia and Dystopia Many biologists/geneticists are in favor of eugenics due to the possibility of advancing the human race, limiting disease, and decreasing the occurrence of negative mutations, while others believe eugenic practices are unethical, useless, and have more potential for harm. Eugenic practices have proven to be extremely controversial, so I will focus on discussing the potential impacts of eugenics on the human body, society, and morality. Modern eugenic practices consist of two types of gene alteration: negative genetic engineering, which is the process of removing genes to combat disease, and gene therapy, which improves one 's genetic make-up (Hix, 2009, para. 4). Both methods of eugenics are equally controversial and equally promising. Gene therapy has been used to
Therefore, if two parents are carriers of a certain gene that will disable their child, they can modify that gene to make a child that will not have that disability. As well as some parents will have designer babies to save another child they already have with a certain disease. In this method, parents will choose their child’s blood type and such in order for them to match that other diseased child and potentially give them their blood, marrow, and even organs. Genetically modifying a child’s chance of disease ensures that a child may live a life without potential disease and disability that they may have been more prone to had their parents’ not used this method. This also ensures a healthy life for a child that had a greater potential of having a medical condition due to their parents being carriers of that particular gene.
The hope for this is that the transplant of stem cells are introduced into the body and replace damaged or dying cells, taking over their function and repairing any tissue damages. Stem cells can also be used in an opposing way to tissue repair, by removing cancerous cells in the body. This attempt is carried out by using the “homing ability” of stem cells as they automatically know to go to a site of damage. Engineered stem cells deliver compounds which are toxic specifically to those tumours, which goes back to them being used for medicinal purposes. In recent years stem cell therapy has been joined with gene therapy.
According to the World Health Organization, genetic disorders affect one in every twenty-five children born worldwide. We humans, along with all other known organisms, possess genes in our cells that determine everything about how we look and function. Occasionally, an organism may inherit a faulty gene, causing it to have one or several detrimental characteristics known as genetic disorders. This may also occur due to environmental factors that the organism is exposed to, such as ultraviolet radiation, which damage its genes and cause alterations in them when they repair. The recent discovery of CRISPR-Cas9, or simply CRISPR, a defense system against viruses in bacteria, has presented the possibility of gene-editing in a way that is faster,
A transgenic organism, or genetically modified organism (GMO), is the organism with the new genome containing the transgene. There are three methods by which transgenic animals can be created: DNA microinjection, retrovirus-mediated gene transfer, and embryonic stem cell-mediated gene transfer. For DNA microinjection, the desired gene must first be created in vitro through the cloning methods described earlier. This DNA is then inserted into the nucleus of an egg ell that has been isolated from an animal. The egg cell is then implanted into a surrogate organism’s oviduct where it can
“The main arguments against genetic modification of human embryos are that it would be unsafe and unfair, and that modification would quickly go beyond efforts to reduce the incidence of inherited maladies” (Caplan). During the altering genes in the mother 's womb cause a lot of dangerous situations and
Gelsinger’s death was a tragedy in the world of science and exposed the nation to the harmful consequences of gene therapy and other genetic manipulations. “Such incidents suggest that the road ahead in treating major diseases - even with knowledge of the human genome - will be far from smooth” (Greenberg). Furthermore, his death reveals the many complications attributed to the many unknowns still present in experiments manipulating the properties of
Gene therapy’s promise to cure the incurable creates mass-support because of the medical appeal however this appeal neglects the ability of the scientific community to follow through. Gene therapy’s public release opposes the global movement for health equality and safety because of the physical inaccessibility, financial barriers, and unpredictable risks. Because gene therapy is only offered in few specific locations, it is inaccessible for a large majority of the world; equality based on physical boundaries has not yet been established for this new treatment. Katelin Peterson et al. in Personalized Medicine, Availability, and Group Disparity describe how gene therapy is more available to certain ethnicities, primarily Caucasian (211).
Stem cell therapies have the potential to be able to regenerate all of the tissues in the human body and along with gene therapy could be the most powerful tools in medicine with the potential to treat a