Cancer Gene Therapy Discussion Essay Paper
Q1
Acute lymphoblastic leukemia (ALL) is a type of cancer affecting the blood and bone marrow, essential parts of the body’s immune system. ALL begins in the bone marrow, where blood cells are formed, and involves the uncontrolled growth of immature white blood cells, known as lymphoblasts (Sheykhhasan, Manoochehri & Dama, 2022). Acute lymphoblastic leukemia leads to pancytopenia, anemia, infections, and bleeding disorders. ALL is the most common type of cancer diagnosed in children below 15 years, accounting for approximately 25% of all pediatric cancers. ALL often arise from poor translocation from chromosome numbers 12 and 21. Other predisposing environmental factors include radiation which damages the bone marrow cells. Cancer Gene Therapy Discussion Essay Paper
ALL often present with massive systemic spread affecting all major lymphatic organs in the body, such as the spleen and liver. The symptoms of ALL can vary but may include fatigue, weakness, bone pain, fever, easy bruising or bleeding, frequent infections, and swollen lymph nodes (Kansagra et al., 2019). The main diagnostic criteria involve the combination of physical exams, blood tests, bone marrow biopsy, and imaging studies. Secondly, the main treatment procedures apply chemotherapy using alkylating drugs like methotrexate and 5-fluorouracil. However, other treatment regime may include radiation therapy, targeted therapy, and bone marrow or stem cell transplantation. Cancer Gene Therapy Discussion Essay Paper
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References
Kansagra, A. J., Frey, N. V., Bar, M., Laetsch, T. W., Carpenter, P. A., Savani, B. N., … & Hashmi, S. K. (2019). Clinical utilization of Chimeric Antigen Receptor T-cells (CAR-T) in B-cell acute lymphoblastic leukemia (ALL)–an expert opinion from the European Society for Blood and Marrow Transplantation (EBMT) and the American Society for Blood and Marrow Transplantation (ASBMT). Bone marrow transplantation, 54(11), 1868-1880.
Sheykhhasan, M., Manoochehri, H., & Dama, P. (2022). Use of CAR T-cell for acute lymphoblastic leukemia (ALL) treatment: a review study. Cancer gene therapy, 29(8-9), 1080-1096.
Q2
Acute renal failure (ARF) is a sudden loss of kidney function that occurs over hours to days. It is characterized by increased serum creatinine and blood urea nitrogen (BUN) levels, waste products excreted by the kidneys (Wang, Chen & Xu, 2020). The exact mechanism of ARF in patients with ALL is that the abnormal lymphoblasts produced in ALL can directly damage the kidneys by infiltrating and damaging the kidney tissue. Direct accumulation and inflammatory damage to the kidneys lead to scarring of the kidneys hence reducing the kidneys’ ability to function correctly.
Secondly, ARF in patients with ALL undergoing chemotherapy may experience the toxic effects of chemotherapy used to treat cancer. Chemotherapy drugs can harm the kidneys and, in some cases, lead to ARF (Wang, Chen & Xu, 2020). Patients with pre-existing kidney disease or receiving high doses of chemotherapy are at a higher risk of developing ARF.
ALL often lead to increased blood viscosity related to ischemic kidney attacks causing acute kidney failures. Secondly, in severe ALL cases, one develops brutal immune suppression leading to increased susceptibility to infections. Increased septicemia and repeated kidney infection may lead to sudden kidney failures from bacteria toxins (Gupta et al., 2020). Dehydration and electrolyte imbalances are more common in patients with ALL. Dehydration complications also contribute to the development of ARF due to impaired perfusion and uric acid excretion. Dehydration also reduces blood flow to the kidneys, leading to kidney damage and dysfunction. Accumulated uric acid in the kidney causes intoxication of kidney cells and cellular deaths. Cancer Gene Therapy Discussion Essay Paper
References
Gupta, S., Seethapathy, H., Strohbehn, I. A., Frigault, M. J., O’Donnell, E. K., Jacobson, C. A., … & Sise, M. E. (2020). Acute kidney injury and electrolyte abnormalities after chimeric antigen receptor T-cell (CAR-T) therapy for diffuse large B-cell lymphoma. American Journal of Kidney Diseases, 76(1), 63-71.
Wang, L., Chen, K., & Xu, Q. (2020). The Application of 99m Tc-DTPA Renal Dynamic Imaging to Measuring Renal Function of Children with Acute Lymphoblastic Leukemia after Induction Therapy. BioMed Research International, 2020.
Q3
Acute sickle cell disease crisis is painful in patients with sickle cell disease (SCD). SCD involves forming rigid and sticky sickle-shaped red blood cells (RBCs), leading to the occlusion of small blood vessels. The occlusion of smaller blood vessels leads to reduced tissues and organ perfusion and death, leading to pain (Darbari, Sheehan & Ballas, 2020). The pain develops from cellular tissue ischemia and hypoxia, and tissue damage.
During acute crises, sickle cells are trapped in small blood vessels, leading to the occlusion of blood flow and tissue ischemia. Ischemia then triggers the activation of the inflammatory response by releasing cytokines, chemokines, and other inflammatory mediators such as bradykinin and interleukins (Alshahrani et al., 2022). Chemical mediators released by ischemic cell then increases the sensitivity of pain receptors and promotes the recruitment of immune cells to the affected area. The predominant feature of acute sickle cell disease crisis is pain due to tissue ischemia and hypoxia (Darbari, Sheehan & Ballas, 2020). The SCD pain is severe, intense, and intermittent all over the body. The SCD pain is also associated with physical stress, infections, dehydration, and temperature change. Cancer Gene Therapy Discussion Essay Paper
References
Alshahrani, M. S., AlSulaibikh, A. H., ElTahan, M. R., AlFaraj, S. Z., Asonto, L. P., AlMulhim, A. A., … & GUIDE Group. (2022). Ketamine administration for acute painful sickle cell crisis: a randomized controlled trial. Academic Emergency Medicine, 29(2), 150-158.
Darbari, D. S., Sheehan, V. A., & Ballas, S. K. (2020). The vaso‐occlusive pain crisis in sickle cell disease: definition, pathophysiology, and management. European journal of haematology, 105(3), 237-246.
Q4
Sickle cell disease (SCD) is a genetic disorder affecting the structure and function of the beta-globin subunit of hemoglobin. A mutation in the HBB gene causes SCD. The mutation causes the replacement of the glutamic acid residue with a valine residue in the beta arm of the hemoglobin chain (Ata et al., 2023). The mutated beta-globin protein forms abnormal sticky, long, and rigid hemoglobin molecules that cause the red blood cells to form the sickle shape. Sickle-shaped red blood cells have a shorter lifespan than normal red blood cells. When trapped in small blood vessels, the sickle cells cause occlusion of blood flow and tissue damage, and pain crisis.
According to Inusa et al. (2019) SCD is an autosomal recessive disorder and only occurs when an inherits two copies of the mutated HBB gene (homozygous inheritance). Heterozygous individuals are carriers of the disease but do not develop symptoms. If two pages have children, there is a 25% chance that each child will inherit two copies of the mutated gene and develop SCD. Similarly, there is a 50% chance that each child will be heterozygous and only be a carrier. Finally, there is an equal chance that 25% chance that each child will inherit two normal copies of the gene and not have the disease or be a carrier. Cancer Gene Therapy Discussion Essay Paper
References
Ata, F., Rahhal, A., Malkawi, L., Iqbal, P., Khamees, I., Alhiyari, M., … & Yassin, M. (2023). Genotypic and Phenotypic Composition of Sickle Cell Disease in the Arab Population-A Systematic Review. Pharmacogenomics and Personalized Medicine, 133-144.
Inusa, B. P., Hsu, L. L., Kohli, N., Patel, A., Ominu-Evbota, K., Anie, K. A., & Atoyebi, W. (2019). Sickle cell disease—genetics, pathophysiology, clinical presentation and treatment. International journal of neonatal screening, 5(2), 20.
Q5
Hemophilia is a congenital bleeding disorder commonly occurring on chromosome X and is marked with deficiency or dysfunction of clotting factors VIII or IX. Hemophilia develops when the platelets cannot stabilize the blood clot leading to uncontrolled bleeding (Leebeek & Miesbach, 2021). During an injury to blood vessels, the body initiates the coagulation cascade to form a blood clot that helps stop the bleeding. The coagulation cascade activates clotting factors that create a fibrin clot. Cancer Gene Therapy Discussion Essay Paper
However, in deficiency or dysfunction of clotting factors VIII or IX among hemophilic patients, the activation activity of fibrin clots is reduced (Leebeek & Miesbach, 2021). Delayed fibrin clot formation leads to prolonged bleeding after injury and idiopathic bleeding of the joints or muscles. A deficiency of factor VIII causes Hemophilia A, while a lack of factor IX causes hemophilia B. Hemophilia A is more common than hemophilia B.
The severity of hemophilia is diagnosed depending on the clotting factor present in the blood. Individuals with severe hemophilia have less than 1% of the average clotting factor level. Severe cases of hemophilia are marked by frequent and massive bleeding. Secondly, individuals with mild hemophilia have more clotting factor concentration, and report reduced bleeding. Hemophilia involves activating compensatory mechanisms such as increased production of fibrinolytic enzymes. High levels of fibrinolytic enzymes cause the breakdown of blood clots and increase vascular endothelial growth factor, promoting the formation of new blood vessels (Petkovic et al., 2022). Angiogenesis among hemophilic patients is associated with many pain crises.
References
Leebeek, F. W., & Miesbach, W. (2021). Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues. Blood, 138(11), 923-931.
Petkovic, M. J., Tran, H. A., Ebeling, P. R., & Zengin, A. (2022). Osteoporosis management and falls prevention in patients with haemophilia: Review of haemophilia guidelines. Haemophilia, 28(3), 388-396.
Please give a response to each question with their reference separately . At least 2 reference is required for each response.
Question #1
Scenario 1: Acute Lymphoblastic Leukemia (ALL)
An 11-year-old boy is brought to the clinic by his parents who states that the boy has not been eating and listless. The mother also notes that he has been easily bruising without trauma as he says he is too tired to go out and play. He says his bones hurt sometimes. Mother states the child has had intermittent fevers that respond to acetaminophen.
Maternal history negative for pre, intra, or post-partum problems.
PMH: Negative. Easily reached developmental milestones.
PE: reveals a thin, very pale child who has bruises on his arms and legs in no particular pattern.
LABS: CBC revealed Hemoglobin of 6.9/dl, hematocrit of 19%, and platelet count of 80,000/mm3. The CMP demonstrated a blood urea nitrogen (BUN) of 34m g/dl and creatinine of 2.9 mg/dl.
DIAGNOSIS: acute leukemia and renal failure and immediately refers the patient to the Emergency Room where a pediatric hematologist has been consulted and is waiting for the boy and his parents.
CONFIRMED DX: acute lymphoblastic leukemia (ALL) was made after extensive testing.
Question
1. Explain what Acute Lymphoblastic Leukemia (ALL) is? Cancer Gene Therapy Discussion Essay Paper
Question #2
Scenario 1: Acute Lymphoblastic Leukemia (ALL)
An 11-year-old boy is brought to the clinic by his parents who states that the boy has not been eating and listless. The mother also notes that he has been easily bruising without trauma as he says he is too tired to go out and play. He says his bones hurt sometimes. Mother states the child has had intermittent fevers that respond to acetaminophen.
Maternal history negative for pre, intra, or post-partum problems.
PMH: Negative. Easily reached developmental milestones.
PE: reveals a thin, very pale child who has bruises on his arms and legs in no particular pattern.
LABS: CBC revealed Hemoglobin of 6.9/dl, hematocrit of 19%, and platelet count of 80,000/mm3. The CMP demonstrated a blood urea nitrogen (BUN) of 34m g/dl and creatinine of 2.9 mg/dl.
DIAGNOSIS: acute leukemia and renal failure and immediately refers the patient to the Emergency Room where a pediatric hematologist has been consulted and is waiting for the boy and his parents.
CONFIRMED DX: acute lymphoblastic leukemia (ALL) was made after extensive testing.
Question
1. Why does ARF occur in some patients with ALL?
Question #3
Scenario 2: Sickle Cell Disease (SCD)
A 15-year-old male with known sickle cell disease (SCD) present to the ER in sickle cell crisis. The patient is crying with pain and states this is the third acute episode he has had in the last 10-months. Both parents are present and appear very anxious and teary eyed. A diagnosis of acute sickle cell crisis was made.
Question
1. Explain the pathophysiology of acute SCD crisis. Why is pain the predominate feature of acute crises? Cancer Gene Therapy Discussion Essay Paper
Question #4
Scenario 2: Sickle Cell Disease (SCD)
A 15-year-old male with known sickle cell disease (SCD) present to the ER in sickle cell crisis. The patient is crying with pain and states this is the third acute episode he has had in the last 10-months. Both parents are present and appear very anxious and teary eyed. A diagnosis of acute sickle cell crisis was made.
Question
1. Discuss the genetic basis for SCD.
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Question #5
Scenario 3: Hemophilia
8-month infant is brought into the office due to a swollen right knee and excessive bruising. The parents have noticed bruising about a month ago but thought the bruising was due to the attempts to crawl. They became concerned when the baby woke up with a swollen knee. Infant up to date on all immunizations, has not had any medical problems since birth and has met all developmental milestones.
FH: negative for any history of bleeding disorders or other major genetic diseases.
PE: within normal limits except for obvious bruising on the extremities and right knee. Knee is swollen but no warmth appreciated. Range of motion of knee limited due to the swelling.
DIAGNOSIS: hemophilia A.
Question
1. What is the pathophysiology of Hemophilia Cancer Gene Therapy Discussion Essay Paper