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NSWBiologyQuick questions

Module 8: Non-infectious Disease and Disorders

Quick questions on Disease management: pharmaceuticals, gene therapy, lifestyle: HSC Biology Module 8

15short Q&A pairs drawn directly from our worked dot-point answer. For full context and worked exam questions, read the parent dot-point page.

What is pharmaceutical intervention?
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Pharmaceuticals modify physiology by targeting receptors, enzymes, transporters or ion channels. They are the workhorse of chronic disease management.
What is gene therapy?
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Gene therapy modifies the patient's DNA to correct a genetic defect. Two main approaches:
What is lifestyle change?
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Lifestyle interventions modify behavioural risk factors and often work on multiple diseases at once.
What are future directions?
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Treating gene therapy as a near-future panacea. It is curative for specific single-gene diseases but unlikely to be widely deployed for complex polygenic disease soon.
What is cancer chemotherapy?
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Cytotoxic agents (cisplatin, doxorubicin, paclitaxel) damage rapidly dividing cells; selective for cancer but with collateral toxicity to bone marrow, gut and hair follicles.
What is gene addition?
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A functional gene is delivered to the patient's cells, usually by a viral vector (adeno-associated virus, lentivirus).
What is gene editing?
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Cas9 nuclease guided by a short RNA precisely cuts a chosen DNA sequence, which the cell repairs through homology-directed repair (introducing a correct sequence) or non-homologous end joining (disabling a gene).
What are strengths?
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Potentially curative; targets root cause; one-time treatment.
What are limitations?
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Cost (Casgevy approximately 3 million Australian dollars per patient); access (specialist centres only); off-target editing risk; ethical concerns around germline editing.
What is physical activity?
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150 minutes of moderate aerobic activity plus 2 sessions of resistance training per week reduces cardiovascular mortality by approximately 30 percent, improves insulin sensitivity, and reduces depression and dementia risk.
What is alcohol reduction?
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Australian guidelines recommend no more than 10 standard drinks per week. Reduction reduces hypertension, atrial fibrillation, liver disease and several cancers.
What is sleep and stress?
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Chronic sleep deprivation and unmanaged stress contribute to hypertension, insulin resistance and depression. Cognitive behavioural therapy is effective for both.
What is q1?
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Distinguish between somatic gene therapy and pharmaceutical intervention for treating a genetic disease. [3 marks]
What is q2?
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A clinical trial of a new statin reports LDL cholesterol falling from 5.2 mmol/L to 2.9 mmol/L over 12 weeks in 300 patients. Calculate the average reduction and explain why this may not translate to reduced cardiovascular events. [3 marks]
What is q3?
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Evaluate the role of gene therapy in treating genetic disease. (a) Identify one approved gene therapy and the disease it treats. (b) Identify one major limitation.

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