Cell Pathology Assignment- Data Analysis 1 -5 pt Pick an organelle from your pos
ID: 140612 • Letter: C
Question
Cell Pathology Assignment- Data Analysis 1 -5 pt Pick an organelle from your poster and look up a particular disorder or disease that is caused by defects in that particular organelle. Describe the symptoms, prognosis and treatment of the disease, and current or recent research topics. How does the function of that organelle play a role in the symptoms and treatment of the disorder? Is this a genetic (inherited disease) or have environmental origins? Be as thorough as you can and use full sentences to answer the questions. List your references with article title, authors, journal, dates and pages. You should have at least 2 full references. Due today by the end of class. TURN in on CANVAS.Explanation / Answer
The organelle which we will be discussing is lungs. The disorder caused in lungs are cystic fibrosis. It's a progressive genetic disease which affects the lungs and digestive system. In this case, a defective gene accumulates mucus in the lungs and digestive tract. Due to the accumulation, bacterial infection occurs and lead to respiratory failure
People with CF can have a variety of symptoms, including:
It is found that cystic fibrosis is caused by genetic disorder an d two genes are involved. One gene is inherited from mother and the other from father. A person with a single gene is known to be carrier
An individual with cystic fibrosis born in the United States today is expected to survive longer than 40 years.The median survival age is higher in males than in females.
With current treatment strategies, 80% of patients should reach adulthood.
The treatments are
1) lung therapy - inhalers, DNase enzyme therapy which cleaves the DNA in the mucus and makes it less spreading, flu and bacterial vaccines, oxygen therapy, lung transplant if needed
2) bowel therapy - maintaining good diet, constipation therapy
The disease is caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which compromises the normal function of the secretory glands. In healthy patients, mucus is a slippery and watery substance, but in CF patients it is sticky and thick
The current research going on in UNSW is Miniaturized versions of each CF patient are created using their own stem cells from lung and gut tissue, called the “CF AVATAR”, which swells up when a potential new drug restores the defective function of the CFTR protein. Notably, a recent study showed the reduction or absence of a novel human gene called BGas was shown to boost CFTR levels independent of CFTR mutation types. We will thus explore the therapeutic potential of a few small molecules targeting BGas in the patient-derived “CF AVATAR”. If successful, gene targeting will herald a new dawn in CF therapy, with one drug that works wonders across the entire CF population
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