Wait a second!
More handpicked essays just for you.
More handpicked essays just for you.
Essay on therapies in hemophilia
Essay on therapies in hemophilia
Essay on therapies in hemophilia
Don’t take our word for it - see why 10 million students trust us with their essay needs.
Recommended: Essay on therapies in hemophilia
A great amount of studies have been conducted on the blood clotting disorder, hemophilia, and it’s plenty of ways to manage the disease. Scientists debate the most effective form of treatment, whether it is doses of factor eight and factor nine injections or gene and cell therapy. Different researchers have provided contradictory evidence to other studies in their quest to find the optimal treatment for hemophilia patients. This paper will address the benefits of each treatment and their efficiency in reducing the patients’ symptoms. Hemophilia patients genetically lack two main coagulation factors, called factor eight (VIII) and nine (IX). Two experts in the hemophilia field, Dr. Hartmann and Dr. Croteau, note current ways to manage the disease can come from factor VIII and factor IX protein replacement through injections (2016). Scientists have debated this method of treatment, although it is the most common, because the proteins have been reported to decrease in their effects over time. It has been proven that “25% of the patients develop inhibitory antibodies to factor VIII following repeated infusions” (Miao, 2016). Over time, …show more content…
Ohmori et al. write that because hemophilia is the results of a gene mutation, it is a perfect fit for treatment through gene and cell therapy (Ohmori et al., 2015). A major advantage of gene therapy is the use of AAV vectors, which are safe and have low risk of falling immune to the body. On the other hands, the treatment of cell therapy, which involves the transplant of cells into the affected persons, has a slight advantage over gene therapy, as it does not include the side effects associated with the AAV vectors. Although it has not been formally experimented, Ohmori believes hematopoietic stem cells can be transplanted and will express as coagulation factors in the blood for a long amount of time, helping relieve hemophilia symptoms in those with the
In some individuals with severe hemophilia, the factor VIII replacement therapy is identified as a foreign substance by their immune system. If this happens, their immune system will make antibodies against factor VIII. These antibodies will inhibit the ability of the factor to work in the clotting process. The higher the antibody or inhibitor level, the more factor VIII replacement therapy it takes to overcome the inhibition and produce clotting. This can complicate the treatment of a bleed. The good news is that there are different types of therapies available to successfully treat most individuals who develop inhibitors.
There have been four somewhat recent successful gene therapy treatments. The four deal with correcting hemophilia, bone marrow transplants, skin cancer, and vessel growth. In the success with the bone marrow transplants, French researchers collected bone marrow cells from patients, used gene therapy to correct the bone marrow, and then returned the bone marrow to the patient. This was 80% successful as reports 16 months after the transplants showed. Squamous cell carcinoma, skin cancer of the head and neck, was treated using gene therapy as well. The fourth trial was where DNA was used to carry a substance that stimulates blood vessel growth to damaged heart tissue and in this trial there was much success noted.
Hemophilia is a rare bleeding disorder that slows the blood clotting process, which is not normal. Some people with Hemophilia may just have a little bit of “clotting factor” or no clotting factor at all (National Institute of Health [NIH], 2013). Clotting factor is a protein in blood that controls bleeding and they are needed the blood to clot normally. In order to help the blood clot, clotting factors work with “platelets” (National Institute of Health [NIH], 2013). Platelets are small blood cell fragments that form in the bone marrow, a tissue in the bones that is similar to a sponge. The functions of platelets have a very important role in blood clotting; the role of a platelet is to stick together (by the help of clotting factors) to block cuts, break on the carriers of blood (veins or arteries) throughout the body, and stop the bleeding when “blood vessels” (National Institutes of Health [NIH], 2013) are injured. Blood vessels are tube like structures carrying blood through the tissues and organs, like a vein, artery, or capillary. People with hemophilia do not have enough “clotting factor VIII or IX” (World Federation of Hemophilia [WFH], 2013) in their blood, which results to prolonged bleeding or oozing, meaning that bleeding can last longer (though, not faster) than usual after surgeries, accidents, or having teeth pulled out at the dentist. Clotting factor VIII, which can also be called as “anti-hemophilic factor” (AHF, for short) (Patient.co.uk, 2011), is a blood clotting protein that is necessary for humans to have. Clotting Factor IX is a protein that i...
...ions over her own body; whether it is parenthood, adoption, contraception, or yes, abortion. After all, it is entirely plausible for people to be personally pro-life but politically pro-choice – this suggests that they respect each individuals’ rights over his or her own body and recognize that each woman should be allowed to have options. Regardless of whether or not she chooses to have the child, it is the fact that she has a choice that is crucial, and it is imperative that each woman have the freedom of individual autonomy. In order for this to occur, abortion must continue to be legal. Ultimately, being pro-choice is not necessarily advocating the use of abortion – it is about ensuring that abortion is legal so that women may have the option to exercise their right to individual autonomy and make the decisions that best reflects their stance in life.
Haemophilia is used to describe a collection of hereditary genetic diseases that affect a mammal’s body’s capability to control thrombogenesis. Thrombogenesis is the way in which blood clots which is an important role in haemostasis. Two common forms of haemophilia are A and B. (1) Someone with A (otherwise known as classic haemophilia), clotting factor VIII is does not exist enough or is entirely absent. A person with haemophilia B (otherwise known as Christmas disease), clotting factor does not exist enough or is also entirely absent. Those with the disorder do not bleed a lot they just simply bleed for a longer period of time. All people with haemophilia A or B are born with the disorder as it is a hereditary disorder and passed down through generations very few cases of haemophilia are not genetic and are therefore rendered a spontaneous gene mutation which is then passed down.
The changes in f8 and f9 gene are responsible for hemophilia A and B. the f8 gene codes for a protein called coagulation 8. This protein is responsible for the blood clotting process. After an injury blood clots protect the body by sealing off the area of injury. Mutation in the f8 and f9 gene can lead to the abnormal form of the protein. Also can lead to a reduction of the coagulation pro...
To conclude, although gene therapy can cure a wide variety of diseases which cannot be cured by traditional medicine, and patients can get permanent cure without rejections, it can be high-risk and immoral. The negative effects of gene therapy lead to the shrink of the number of volunteers, and many trials have been forced to cease. The Gene therapy's potential to revolutionize medicine in the future is exciting, and hopes are high for its role in curing and preventing childhood diseases.
Abortion has been the topic of controversy for many decades. Many people believe that when a woman terminates a pregnancy, she is committing murder and others argue that a woman has the right to choose life or abortion. There are different procedures to choose when having an abortion, depending on the gestational age and the woman 's health a pill form abortion may be used up to 9 weeks gestation (mifepristone and misoprostol), but for women who are over 12-weeks gestation (late-term abortion), surgical abortion is used (Berer 25). In 1973, the supreme court ruled that abortion was to be legalized, Roe vs. Wade. Women were given the legal right to choose to terminate their pregnancies and make the correct arrangements for their decisions. Different states have different restrictions to accessing abortion procedures, making the woman 's choice to terminate pregnancy less accessible. Restricting a woman 's access to appropriate abortion clinics limits her right to choose.
The birth of a child is usually a wonderful and priceless occasion. However, on June 5, 2015, an eleven-year-old girl gave birth to a newborn girl. Approximately a year before she gave birth, her 40-year-old father repeatedly sexually assaulted her. In this case, the unprepared eleven-year-old child decided to have the baby. This is a prime example that illustrates that the right to abortion should always be vested in the woman. Abortion, which has been debated for centuries and will continue to be fought upon for centuries to come, is a hot issue among social, political, and religious entities. This research paper will inform the reader about abortion using scholarly journals to define abortion, specifically when a fetus becomes a human being,
Imagine if it were you: missing school or work since you cannot afford the products you need. Imagine if it were you: forced to leave your home as it is "that time of the month". Imagine if it were you: shunned by family and friends; made to feel like less of a person simply because of a natural bodily function. This is life for millions of women around the world and it needs to change. Women are historically encouraged to hide their periods. Even today, in "modern" society, menstruation is still highly stigmatized. This stigmatization leads to serious problems with health, social situations, personal development, society, and even endangers women's lives.
As a young adult, it may seem foolish to predict what your future family life will look like, especially in regards to children. Often times this reality is forced upon a select few, particularly homosexual couples; however, with the innovation of in vitro fertilization (IVF), a couple is met with promise and the hope of a successful family life. IVF can be described as a process by which a fetus is genetically formed in a laboratory setting. Though this process may seem unnatural in essence, it allows for a more diverse family arrangement through medical innovation. This procedure, though controversial, is seen by many as an advancement in the medical field and can be accredited to procuring a healthy child for an unfortunate family, whether
Polycythaemia is a blood disorder defined as an increase in blood erythrocyte concentration. Absolute polycythaemia is where this increase is caused by greater erythrocyte production, determined by measuring the haematocrit level, with one study showing that 83% of sufferers have a haematocrit level of >55%. This should not be confused with relative polycythaemia, caused by a decrease in blood plasma volume often secondary to hypertension. Interestingly, those affected tend to also have increased blood platelet and white cell concentrations, which correlates well with the notion that the disease is caused by a genetic defect in the haematopoietic stem cell population within the bone marrow. Studies have shown the median age of onset to be 60, although a Mayo Clinic study in Olmstead County, Minnesota showed it to be slightly higher, at between 70 and 79 years, with men affected more than women – though the reason behind the gender inequality is currently unknown. In addition, its prevalence within the US is thought to be in the region of 50/100,000. Furthermore, it has long been known that Finnish skier and seven time Olympic champion Eero Mantyranta suffered from the disease, and it is often stated that his success, at least in part, can be put down to his polycythaemia – indeed experiments have shown that it can increase the blood oxygen carrying capacity by up to 50%, an undeniable asset to any endurance athlete. However, the disease can also prove fatal in many cases because the thickened blood increases the probability of clot formation, giving deep vein thrombosis and potentially pulmonary embolus. It is this double edged nature of polycythaemia that first took ...
The first successful case of stem cell therapy in human was reported in 1959. Bone marrow restorations were observed in leukemia patients who received total body irradiation subsequent by intravenous injection of their twins’ bone marrow (Thomas et al, 1957). Nevertheless, that effect was transient and the following bone marrow transplantation attempts in non-twin patients and donors can eventually lead to patient’s death from graft-versus-host disease (Mathé et al, 1965). During that time, the safety of hematopoietic cells transplantation was not guaranteed because of the limited knowledge in human histocompatibility and immunosuppression. However, the turning point came after the discovery of human leucocyte antigen (HLA) groups (Dausset, 1958; van Rood et al, 1958), HLA typing and compatibility testing were performed prior to the transplantation. In addition, the improvement of immunosuppressive protocol also helps bringing the bone marrow transplantation to become more and more successful (Donnall and Hutchinson, 1999).
Abortion Taking out the trash, or taking a human life? That is what most the argument is with abortion, some believe that abortion is okay because the fetus is not yet a person, and that the fetus will be nothing but trouble. On the other side people believe that even all the way down to the zygote (when sperm meets the egg and earliest stage of pregnancy) there is a human growing in you. A growing boy or girl, with their own little fingers, and toes, their own cute as can be toothless smile, and their functioning organs. To them one day that zygote will turn into someone great, a person just like you and I that needs love just as you and I, a person that deserves to not get taken from our world before it even has a chance to take its first breath.
Abortion has been one of the most talked about topics in society just about anywhere from television, magazines, whether or not it should be the right or wrong thing to do. Abortion is a very sensitive issue to discuss, because of its nature. Many people have said that abortion is a very bad thing to do and it should not even be choice whether or not to abort a living fetus. People think that abortion is committing murder as it is killing the human fetus. However, others feel that a woman should have a voice and have the right to choose to keep the child or not and that it is not murder until the baby is born. Majority of individuals who believe that abortion is bad say that the fetus is human who is partly being formed and to have an abortion is considered to be murder. For the people who think an abortion is ok, say that it’s not considered murder unless the child is born. I believe that abortion should be seen in which the stage the fetus is in. if the fetus is in an early stage of pregnancy it is not considered murder, but if the fetus has already began to develop into a larger fetus then it is indeed considered to be murder. There are times when abortion can be accepted, if the mother is having complications due to pregnancy. For example if the mother is enduring complications in her pregnancy that can harm her, because of the child in that case it is ok to perform an abortion to help save the mother’s life. It is also very important to understand this type of situation. The mother has the right to have an abortion and it is her decision because a mother knows best about her health conditions.