Severe Combined Immunodeficiency Severe Combined Immunodeficiency (SCID) is a primary immune deficiency, which is a disorder caused by an inherited flaw in the immune system that increases the chance of getting diseases for a person. The most distinctive characteristic of SCID is a defect in both the T- & B-lymphocyte systems which are vital parts of a immune system . This will result in with one or more infections that are deadly for babies within the few months of life. Children who have SCID can also become ill from live viruses that are in some vaccines. These vaccines have viruses and bacteria that are weakened, but don’t harm children who are healthy. People with SCID however, these viruses and bacteria can cause life-threatening infections.(SCID, …show more content…
The first gene therapy trial was started by Dr.William French Anderson. And the patient was Ashanthi,a four year old girl who had SCID. Ashanthi had the disease because of the missing enzyme adenosine deaminase (ADA). This missing ADA stopped her body from constantly producing white blood cells that are needed to fight infections. Ashanthi was in a very vulnerable state with infections that are seen as mild. Ashanthi was able to get two choices of treatment, Antimicrobial drugs that can be used to treat SCID but only with short term benefits or a bone marrow transplant from a matching donor. The bone marrow is a soft tissue located in the centre of our bones and its purpose is to make red blood cells, platelets and white blood cells. For Ashanthi, a transplanted bone marrow would increase the production of white blood cells and would give her a strong immune system to fight the infections. Sadly however, this option was not successful because of the lack of compatible bone marrow donors. Then scientists decided to start a new gene therapy to treat Ashanthi. The scientists would get Ashanthi’s white blood cells from her blood and working copies of the adenosine deaminase gene were put into the cells by a vector. A vector is a “vehicle” used by scientists to add new genes into the DNA, But for Ashanthi, the
According to the CDC, all vaccines carry a risk of a life-threatening allergic reaction (anaphylaxis) in about one per million children. Most vaccine-preventable diseases can cause serious or life-threatening infections in infants and young children. For example, exposure and infection with polio can occur at a very young age and can cause paralysis, so the vaccine should be given to infants as soon as possible. People should be aware of what vaccines are capable to do other than save lives, because of the life threatening effects, the harmful ingredients, and vaccines are unnatural.
It was in the 1980’s that scientist began looking at alternative ways of treatments, one is gene therapy. Scientist would insert human genes into a bacteria cell. Then the bacteria cell would transcribe and translate the information into a protein. Once that is done the scientist would then introduce the protein into human cells. Gene therapy can be simply viewed as inserting bits of foreign DNA into a patient’s tissue in hope...
Vaccines are becoming increasingly hazardous for many children and parents are not being informed about the safety of their children. Current reports are linking vaccines to serious life-threatening disorders such as asthma, autism, immune system dysfunction, and mental retardation (Williams). These recent revelations are causing an increasing amount of people to claim religious and medical exemptions from vaccines. From 1999 to 2006, exemptions have more than doubled from 9,722 to 24,919 (Cronin). It is very clear that vaccinations are posing many problems for parents everywhere. Each day researchers are finding out about vaccines and are realizing that there are a lot more risks than benefits. Dr Phillip F. Incao explains: “Today, far more children suffer from allergies and other chronic immune system disorders than from life-threatening infectious disease. It is neither reasonable nor prudent to persist in presuming that the benefits of any vaccination outweigh its risk” (qtd in Spaker). While infectious diseases are becoming uncommon there is no need for any person to get vaccinated.
Human gene therapy is a method used in the medical field that treats diseases at a molecular level, by solving the source of the problem; our genes. Today, diseases and disorders are commonly treated by solving the symptoms, the surface of the problem. Many disorders and diseases are caused by defective proteins and within those defective proteins are damaged and defective genes. These defective genes can be treated through gene therapy. Gene therapy is not new and has been developed and improved by researchers for the past couple years. Being an experimental technique, gene therapy also has its pros and cons, but so far is showing positive and rising success rates.
David Vetter was the original “Bubble Boy”, he lived for twelve years and made the treatment of other young children possible. Scientists were able to use David’s blood cells to help in the treatment of 16 other kids suffering from SCID. Nine years later 14 of the 16 are leading healthy lives, outside the bubble. David died from lymphoma after receiving a bone marrow transplant from his sister. One of the other children contracted treatment-related leukemia, which wasn’t a shock because several children in France contracted leukemia after getting gene therapy.
Over 40 years ago, two men by the names of James Watson and Francis Crick discovered deoxyribonucleic acid, or DNA. DNA is hereditary material in humans and almost all other organisms (What is DNA?). From this finding, gene therapy evolved. Today, researchers are able to isolate certain specific genes, repair them, and use them to help cure diseases such as cystic fibrosis and hemophilia. However, as great as this sounds, there are numerous ethical and scientific issues that will arise because of religion and safety.
Gene therapy gives people who suffer from genetic diseases a chance to lead a normal life. Dangerous diseases, such as AIDS, SCID, Thalassemia and ADA can be cured successfully. In September 5, 2006, two people with advanced melanoma received Gene therapy and they got recovery soon. This is a breakthrough in cancer gene therapy. Gene therapy uses patients own cells to cure diseases, and, therefore, no rejection to their bodies. Furthermore, patients could get permanent cure from gene therapy without recurrence.
It was a treatment for a four-year-old girl named Ashanthi DeSilva, who were born with an adenosine deaminase (ADA) deficiency, an autosomal recessive disorder that affect the immune system. Her doctors genetically modified her defective immune cells to function as normal ones. Then, they used a virus that also had been genetically modified to remove its harmful genes to deliver the corrected immune cells back to her body. This early success led to many other gene therapy trials in the 1990s for different kinds of genetic diseases, until a tragic setback happened. In September 1999, Jesse Gelsinger became the first person who died after undergone a gene therapy for ornithine transcarbamylase (OTC) deficiency, a rare metabolic disorder. He died from massive organ failure caused by a bad reaction of his immune system to the virus used in the therapy (Thompson
Vaccines have prompted some of the best public health triumphs continually, including the annihilation of smallpox from the globe and the close extermination of polio. They are the organisation of antigenic material to produce immunity to a disease, ameliorating the impacts of infection by a pathogen. The material administrated can either be live, yet debilitated types of pathogens such as bacteria or viruses, executed or inactivated, or refined material such as proteins. Little children are less resistant to diseases a couple of months after birth; it is therefore easier for them to become infected with a disease being passed around if they attend childcare with other children who might not be vaccinated. The Australian government should undoubtedly be able to prevent unvaccinated children from attending childcare centres; however, exceptions ought to be made for children unable to vaccinate due to undergoing treatment that compromises their immune system i.e. chemotherapy, or severe allergies to certain ingredients in vaccines.
Stratton, Kathleen, Wilson, Christopher & McCormick, Marie. (2002). Under Review: Multiple Immunizations and Immune Dysfunction. Immunization Safety Review. (Pgs. 32-42). Retrieved from http://www.nap.edu/openbook.php?record_id=10306&page=32
This means children receive more than one vaccination for every time they arrive at the pediatrics office. Many parents question if too many vaccinations overwhelm a child’s immune system. According to the Center for Disease Control and Prevention (CDC), no evidence suggests that the recommended childhood vaccinations can “overload” the immune system. In contrast, when infants are born, they are exposed to bacteria and viruses. Eating food introduces new bacteria to the digestive system. Most microorganisms cause no problems. Many found in the digestive system produce life- sustaining nutrients, which are essential for good health. When infants play with toys, they are also introducing new bacteria and viruses to their body. According to Brody, when a child puts things in their mouth, they are allowing their immune system to explore the environment. Not only does this allow for practice of the immune responses, but it plays a crucial role in teaching the mature immune system response to what is best ignored. Infant immune systems easily handle weakened or killed vaccine antigens (immunization). Therefore, vaccinations are little compared to what children face every
Severe combined immunodeficiency (also known as SCID) is an uncommon genetic disorder. SCID drastically affects the immune system, harming the T and B cell functions (Severe combined immunodeficiency). More people tend to have X-linked SCID. Males only have one X chromosome and one Y chromosome. Mothers would pass their X chromosomes to their sons. In X-linked SCID, a mother with a defective X chromosome for SCID would pass this gene onto her son, since he only has one X chromosome (Severe combined immunodeficiency). Thus, males have a higher chance of getting X-linked SCID than females (females have two X chromosomes, so if only one of the X chromosomes has the defective copy of the SCID gene, then the female would probably be a carrier for
...matopoietic compartment using integrating vectors particularly need to understand genotoxicity risks in relation to the risks of conventional bone marrow transplantation. A QPL could direct them to ask questions about risk, benefits and survival rates following transplantation at local centres; the prognosis of patients in the different haematopoietic gene therapy trials; the number and status of patients that developed leukaemia in the SCID-X1 gene therapy trials; and whether there are any differences between the proposed vector and the vector used in the SCID-X1 trial and any possible safety developments. This kind of guidance may help patients understand both what is known and unknown about specific applications of gene therapy.
Gene therapy enables patients to survive incurable diseases. In the field of genetic diseases, ADA-SCID, CGD and hemophilia are three main ones. ADA-SCID is known as the bubble boy disease. CGD is related to immune system that would lead to fungal infections which are fatal. Patients with Hemophilia are not able to induce bold bleeding (Gene therapy for diseases, 2011). Gene therapy also has good effects on cancer treatment and neurodegenerative diseases, which include Parkinson’s disease and Huntington’s disease. Viral infections, including influenza, HIV and hepatitis can also be treats by it (Gene therapy for diseases, 2011). According to the Science Daily in 2011, gene therapy now can apply to heart failures and neurologic diseases as well.
“Childhood vaccines are one of the great triumphs of modern medicine. Indeed, parents whose children are vaccinated no longer have to worry about their child's death or disability from whooping cough, polio, diphtheria, hepatitis, or a host of other infections.” (Ezekiel J. Emanuel, 1). Vaccines helped humanity for many years in eliminating illnesses that disfigured, disabled and a lot of times took lives away. Children who do not get vaccinated not only risk themselves by being an easy target for diseases they also, harm everyone around them. In the end, today's children are the fuel of the future. Every parent should think carefully before taking any chance that may harm the coming generation.