L2. Recombinant Therapeutic Proteins I - AI MCQs
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Which statement about the function of rhDNase I in cystic fibrosis therapy is correct?

  • rhDNase I works by increasing the size of DNA fragments in the airways.
  • rhDNase I reduces mucus viscosity by degrading DNA into smaller fragments. (correct)
  • rhDNase I is ineffective in reducing recurrent respiratory exacerbations.
  • rhDNase I is administered systemically to improve the immune response.

    • Which mechanism does rhDNase I primarily use to enhance lung function in cystic fibrosis patients?

  • By acting as an antibiotic to eliminate bacterial infections in the lungs.
  • By directly stimulating ciliary movement within the airways.
  • By increasing mucus production to trap more bacteria.
  • By reducing the viscoelasticity of the sputum through DNA degradation. (correct)

    • Which of the following is an incorrect statement regarding the approval and use of rhDNase I since its FDA approval?

  • The majority of cystic fibrosis patients currently use rhDNase I therapy.
  • rhDNase I was approved in 1993 and has increased in clinical use since then.
  • rhDNase I therapy is linked to improved mucus clearance and reduced respiratory symptoms.
  • rhDNase I provides permanent resolution of lung infections in cystic fibrosis patients. (correct)

    • Which of the following accurately describes the relationship between rhDNase I concentration and its therapeutic effects?

    <p>A direct relationship exists between rhDNase I concentration and the reduction of sputum viscosity.</p> Signup and view all the answers

    Which statement about the biological activity of rhDNase I is incorrect?

    <p>rhDNase I works by increasing the size of DNA to improve airway clearance.</p> Signup and view all the answers

    Which of the following statements about the role of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) is correct?

    <p>CFTR assists in maintaining the balance of salt and water on epithelial surfaces.</p> Signup and view all the answers

    In cystic fibrosis, what is the consequence of the mutation in the CFTR gene?

    <p>Chloride ions are not secreted and remain inside the cell.</p> Signup and view all the answers

    Which of the following correctly describes the effect of sodium ions in cells impacted by cystic fibrosis?

    <p>Sodium ions remain with chloride ions, leading to decreased osmotic pressure.</p> Signup and view all the answers

    Which statement about the physical properties of secretions in cystic fibrosis is incorrect?

    <p>The viscosity of secretions in cystic fibrosis is the result of excessive water loss.</p> Signup and view all the answers

    Which of the following is a direct effect of the CFTR mutation on lung function?

    <p>Increased susceptibility to infections due to thick secretions.</p> Signup and view all the answers

    What physiological mechanism is disrupted due to the malfunction of CFTR in cystic fibrosis?

    <p>Osmotic regulation due to chloride ion balance.</p> Signup and view all the answers

    Which statement regarding the overall impact of cystic fibrosis on the exocrine glands is correct?

    <p>The secretions become thicker, impacting gland function.</p> Signup and view all the answers

    What happens to water movement in cells affected by cystic fibrosis due to CFTR dysfunction?

    <p>Water follows sodium ions, leading to dehydration of surrounding tissues.</p> Signup and view all the answers

    Which statement about recombinant proteins is correct?

    <p>Their production requires the use of common bacterium's genetic material.</p> Signup and view all the answers

    Which of the following is an incorrect statement about cystic fibrosis?

    <p>It is a condition primarily affecting the immune system.</p> Signup and view all the answers

    Which of the following statements regarding the approval of recombinant proteins by the US FDA is incorrect?

    <p>All recombinant proteins have a therapeutic use.</p> Signup and view all the answers

    Which option incorrectly describes the genetic engineering technique in recombinant protein production?

    <p>It requires the manipulation of viral genomes.</p> Signup and view all the answers

    Which statement about the role of recombinant proteins in biomedicine is incorrect?

    <p>They are primarily used in the diagnosis of genetic diseases.</p> Signup and view all the answers

    Which of the following statements about genetic conditions is incorrect?

    <p>All genetic conditions lead to visible symptoms from birth.</p> Signup and view all the answers

    Which statement regarding the movement of salt and water in cystic fibrosis sufferers is true?

    <p>It disrupts normal cellular transport functions.</p> Signup and view all the answers

    Which of the following is an incorrect way to describe the recombinant protein industry?

    <p>Recombinant proteins are only used for rare diseases.</p> Signup and view all the answers

    Which statement about the production of recombinant proteins is incorrect?

    <p>It is limited to bacterial systems for recombinant drug production.</p> Signup and view all the answers

    Which statement about recombinant therapeutic proteins is correct?

    <p>They can be used to address genetic disorders.</p> Signup and view all the answers

    Which of the following describes a mechanism of action for recombinant insulin?

    <p>Stimulation of glucose uptake by muscle cells.</p> Signup and view all the answers

    Regarding the therapeutic applications of rhDNase, which statement is incorrect?

    <p>It is used primarily for the treatment of diabetes mellitus.</p> Signup and view all the answers

    Which claim about the types of recombinant insulin is true?

    <p>Rapid-acting insulin is effective for meal-time glucose control.</p> Signup and view all the answers

    Which of the following statements regarding cystic fibrosis is incorrect?

    <p>Recombinant proteins have no effect on its treatment.</p> Signup and view all the answers

    Which statement related to diabetes management and recombinant insulin is false?

    <p>Insulin has no impact on blood glucose levels.</p> Signup and view all the answers

    Which statement about genetic factors in cystic fibrosis is accurate?

    <p>Genetic mutations in the CFTR gene lead to clinical symptoms.</p> Signup and view all the answers

    Which of the following describes the impact of recombinant therapeutic proteins on disease pathways inaccurately?

    <p>They create completely new proteins not existing in nature.</p> Signup and view all the answers

    Which is a false statement regarding the application of recombinant therapeutic proteins?

    <p>They are only used in severe forms of genetic diseases.</p> Signup and view all the answers

    Which of the following is an incorrect statement about the role of recombinant proteins in diabetes management?

    <p>Their primary function is to prevent insulin resistance.</p> Signup and view all the answers

    Which of the following accurately describes the impact of CFTR genetic mutation on mucociliary clearance?

    <p>It results in impaired mucociliary clearance due to altered airway secretions.</p> Signup and view all the answers

    Which of the following complications is NOT associated with cystic fibrosis?

    <p>Regulated neutrophil response in the airways.</p> Signup and view all the answers

    Which of the following statements about bacterial growth phases is incorrect in relation to cystic fibrosis?

    <p>The decline phase is characterized by nutrient abundance and optimal growth.</p> Signup and view all the answers

    Which statement about neutrophil-dominated inflammation in cystic fibrosis is correct?

    <p>It contributes to airway damage and infection proliferation.</p> Signup and view all the answers

    Which of the following conditions is a direct result of the CFTR protein's malfunction?

    <p>Thick, viscous secretions causing lung obstruction.</p> Signup and view all the answers

    Which of these is an incorrect association between cystic fibrosis and its complications?

    <p>Frequent respiratory infections leading to improved lung capacity.</p> Signup and view all the answers

    Which of the following best describes the consequence of ineffective neutrophil action in cystic fibrosis?

    <p>Accumulation of extracellular DNA causing mucus obstruction.</p> Signup and view all the answers

    Which consequence of cystic fibrosis could potentially lead to premature death?

    <p>Early onset of chronic lung infections.</p> Signup and view all the answers

    Which of the following effects of cystic fibrosis is erroneously identified?

    <p>Infection-free lungs in early childhood.</p> Signup and view all the answers

    Which of the following consequences of poorly regulated inflammation is incorrect?

    <p>Enhanced lung tissue regeneration over time.</p> Signup and view all the answers

    Which of the following statements accurately describes the role of rhDNase I in improving lung function for cystic fibrosis patients?

    <p>rhDNase I reduces mucus viscosity by lowering the molecular weight of DNA fragments.</p> Signup and view all the answers

    Which of the following mechanisms regarding the effect of rhDNase I on airway mucus is incorrect?

    <p>Aerosolized rhDNase I directly increases mucin secretion from goblet cells.</p> Signup and view all the answers

    Which of the following statements about the approval and clinical use of rhDNase I since 1993 is inaccurate?

    <p>rhDNase I was approved for use due to its ability to degrade proteins in the mucus.</p> Signup and view all the answers

    Which statement regarding the relationship between rhDNase I concentration and its therapeutic effects is incorrect?

    <p>Low doses of rhDNase I are sufficient for optimal therapeutic effects.</p> Signup and view all the answers

    Which of the following statements about the reduction of viscoelasticity by rhDNase I is incorrect?

    <p>Reductions in viscoelasticity do not influence the deposition of bacterial infections in the airways.</p> Signup and view all the answers

    Which statement regarding the movement of chloride ions in individuals with cystic fibrosis is correct?

    <p>Chloride ions remain trapped in the cells along with sodium ions.</p> Signup and view all the answers

    Which of the following statements about the role of CFTR in maintaining fluid balance is incorrect?

    <p>CFTR mutation leads to reduced sodium retention in exocrine glands.</p> Signup and view all the answers

    Which of the following is a direct consequence of the mutation in the CFTR gene?

    <p>Thicker and more viscous secretions in exocrine glands.</p> Signup and view all the answers

    In cystic fibrosis, which of the following correctly describes the osmotic result of sodium ion presence in the cells?

    <p>Water follows sodium ions into the cells by osmosis.</p> Signup and view all the answers

    Which statement about the composition of secretions in individuals with cystic fibrosis is incorrect?

    <p>Secretions typically contain elevated levels of chloride ions.</p> Signup and view all the answers

    Which of the following accurately describes the overall impact of CFTR mutations on mucosal surfaces?

    <p>Mucosal surfaces experiences impaired clearance of pathogens.</p> Signup and view all the answers

    Which of the following outcomes is directly associated with the dysfunction of CFTR protein?

    <p>Development of chronic respiratory infections.</p> Signup and view all the answers

    Which statement regarding the physiological mechanisms affected by cystic fibrosis is correct?

    <p>Chloride transport disruption leads to osmotic imbalances.</p> Signup and view all the answers

    Which of the following statements about recombinant insulin in diabetes management is correct?

    <p>Recombinant insulin is designed to mimic natural insulin secretion patterns.</p> Signup and view all the answers

    Which statement regarding the role of recombinant proteins in treating cystic fibrosis is incorrect?

    <p>The use of recombinant proteins has no effect on inflammation associated with cystic fibrosis.</p> Signup and view all the answers

    Which of the following correctly describes the association between genetic factors and cystic fibrosis?

    <p>The CFTR gene mutations lead to dysfunctional chloride channels.</p> Signup and view all the answers

    Which statement about the effects of rhDNase in cystic fibrosis therapy is incorrect?

    <p>rhDNase directly addresses the ion imbalance caused by the CFTR mutation.</p> Signup and view all the answers

    Which of the following statements regarding types of recombinant insulin is incorrect?

    <p>Intermediate-acting insulin has the fastest onset of action among all types.</p> Signup and view all the answers

    Which statement about the biological activity of recombinant proteins is incorrect?

    <p>Recombinant proteins are used primarily for antibiotic treatment.</p> Signup and view all the answers

    Which statement regarding the genetic challenges in diabetes management is correct?

    <p>Type 2 diabetes is influenced by both environmental and genetic factors.</p> Signup and view all the answers

    Which of the following statements about the mechanisms of action of therapeutic proteins is incorrect?

    <p>Therapeutic proteins provide a permanent fix to genetic disorders.</p> Signup and view all the answers

    Which statement regarding the relationship between recombinant proteins and disease pathways is correct?

    <p>Therapeutic proteins generally interact with well-defined biochemical pathways.</p> Signup and view all the answers

    Which of the following accurately describes the result of CFTR genetic mutation on airway function?

    <p>Poorly regulated inflammation contributes to airway damage.</p> Signup and view all the answers

    Which statement regarding the relationship between bacterial infection and cystic fibrosis is incorrect?

    <p>Bacterial growth is unaffected by the thick mucus present in cystic fibrosis.</p> Signup and view all the answers

    Which of the following statements about neutrophil activity in cystic fibrosis is correct?

    <p>Excess neutrophil accumulation leads to airway damage.</p> Signup and view all the answers

    Which consequence of CFTR mutation is inaccurately described among the options?

    <p>Reduction in the viscosity of mucous secretions.</p> Signup and view all the answers

    Which of the following statements about the impact of cystic fibrosis on nutritional health is inaccurate?

    <p>Nutritional deficiencies are solely due to decreased food intake.</p> Signup and view all the answers

    Which of the following correctly identifies a complication related to cystic fibrosis?

    <p>Regular lung infections lead to chronic conditions.</p> Signup and view all the answers

    Which statement about the progression of cystic fibrosis complications is false?

    <p>Excessive inflammation contributes to lung health over time.</p> Signup and view all the answers

    Which statement accurately describes the process of creating recombinant proteins?

    <p>Bacteria can be genetically engineered to produce human proteins after gene insertion.</p> Signup and view all the answers

    Which of the following correctly characterizes the impact of CFTR malfunction on organ systems?

    <p>Both lungs and pancreas can incur damage due to CFTR malfunction.</p> Signup and view all the answers

    Which of the following is an inaccurate statement regarding cystic fibrosis?

    <p>The primary cause of cystic fibrosis is environmental factors.</p> Signup and view all the answers

    Which statement about poor mucosal clearance in cystic fibrosis is inaccurate?

    <p>It causes decreased airway obstruction over time.</p> Signup and view all the answers

    Which statement regarding the role of recombinant proteins in disease treatment is incorrect?

    <p>All recombinant therapeutic proteins have equal efficacy across different diseases.</p> Signup and view all the answers

    Which of the following statements related to the role of inflammation in cystic fibrosis is correct?

    <p>Episodic inflammation accelerates lung damage.</p> Signup and view all the answers

    Which of the following statements about genetic engineering in recombinant protein production is incorrect?

    <p>Only viral vectors are used for introducing human genes in bacteria.</p> Signup and view all the answers

    Which statement about the FDA approval of recombinant proteins is false?

    <p>Only a few, less than 10, recombinant proteins have received FDA approval.</p> Signup and view all the answers

    Which statement regarding cystic fibrosis and its mechanism is incorrect?

    <p>The disease primarily affects the kidneys and liver.</p> Signup and view all the answers

    Which of the following statements about recombinant insulin is true?

    <p>It has transformed diabetes management since its introduction.</p> Signup and view all the answers

    Which of the following accurately describes a major consequence of CFTR dysfunction in cystic fibrosis?

    <p>Accumulation of thick mucus leading to infections.</p> Signup and view all the answers

    Which statement about the commercial success of recombinant proteins is incorrect?

    <p>Pharmaceuticals for recombinant proteins are primarily sourced from plant-based systems.</p> Signup and view all the answers

    Which of the following statements about cystic fibrosis symptoms is incorrect?

    <p>Skin dryness is a common symptom in cystic fibrosis.</p> Signup and view all the answers

    Study Notes

    Course Information

    • Course title: Biochemistry and Biotechnology Fundamentals Recombinant Therapeutic Proteins I
    • Course code: 1120-111
    • Instructor: Dr. Ahmed Hemdan
    • Department: Pharmaceutical Chemistry

    Contents

    • Cystic Fibrosis
    • rhDNase
    • Type 1 diabetes mellitus
    • Insulin
    • Recombinant Insulin

    Learning Objectives

    • Explain the impact of recombinant therapeutic proteins in treating cystic fibrosis and diabetes mellitus
    • Describe the mechanisms of action for therapeutic proteins like rhDNase and recombinant insulin, including interactions with disease pathways
    • Differentiate between types of recombinant insulin, their applications, and benefits for diabetes management
    • Discuss the genetic factors contributing to cystic fibrosis and diabetes mellitus, and how recombinant proteins address these challenges

    Recombinant Proteins

    • Recombinant proteins are artificially produced using genetic engineering techniques
    • They play a critical role in biomedical biotechnology, as research tools and drugs for various diseases
    • The process involves inserting a human gene into the genetic material of a common bacterium, producing the desired protein
    • Recombinant human insulin, produced in 1982, was the first recombinant protein used in treatment
    • Over 130 recombinant proteins are approved for clinical use by the US FDA

    Cystic Fibrosis

    • Cystic fibrosis is a genetic condition caused by a faulty gene affecting salt and water movement in and out of cells
    • Recurrent infections result in thick, sticky mucus buildup in the lungs and digestive system
    • The faulty gene is the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator)
    • CFTR regulates Cl- permeability in exocrine glands, maintaining salt and water balance
    • Mutations in the CFTR gene can result in a build-up of mucus, affecting the lungs and other organs

    Cystic Fibrosis: Key Cellular Processes

    • Cl- is not secreted, staying with Na+ in the cell; H2O follows Na+ leading to thick, viscous secretions
    • Primarily affects the lungs and pancreas, leading to mucus blocks in airways and pancreatic ducts

    Cystic Fibrosis: Complications

    • Worsening lung function: impacting daily activities
    • Lung infections
    • Pancreatic inflammation
    • Vitamin deficiencies (fat-soluble vitamins)
    • Fat in the stools
    • Thick mucus obstructing organs: causing malnutrition, poor growth, respiratory problems, chronic lung disease, and gallstones

    Deoxyribonuclease (DNase)

    • A type of glycoprotein endonuclease enzyme
    • Catalyzes the cleavage of phosphodiester linkages in DNA, degrading it
    • Breaks down extracellular DNA produced by apoptosis and neutrophil extracellular necrosis
    • Reduced inflammation, a key part of cystic fibrosis treatment
    • Used as a treatment in diseases caused by extracellular DNA, such as cystic fibrosis

    Recombinant Human DNase

    • rhDNase is an enzyme that breaks down DNA strands in airway secretions of cystic fibrosis patients
    • Reduces mucus viscosity in the lungs which promotes secretion clearance
    • Applied to treat cystic fibrosis

    Recombinant Human DNase: Mechanism of Action

    • Aerosolized rhDNase degrades DNA in airway secretions into smaller molecules
    • Reduced mucus viscosity allows for expectoration
    • Improves lung function and lowers bacterial infection rate

    Recombinant Human DNase: Clinical Use

    • Cloned, sequenced, and expressed recombinantly using mammalian cell culture
    • In vitro studies on CF patients' sputum show reduced viscoelasticity, directly linked to rhDNase concentration and reduction in DNA size
    • Improved pulmonary function and reduced infections

    Type 1 Diabetes Mellitus

    • A chronic condition, once known as juvenile or insulin-dependent diabetes
    • The pancreas produces little to no insulin
    • Insulin is a hormone allowing sugar (glucose) into cells for energy production
    • Caused by genetics and some viruses
    • Usually starts in childhood or adolescence, but can develop in adults
    • Currently incurable
    • Treatment focuses on managing blood sugar levels through insulin, diet, and lifestyle changes

    Type 1 Diabetes Mellitus: Complications

    Diabetic nephropathy, peripheral neuropathy, eye injury, diabetic retinopathy, kidney disease, cardiomyopathy, foot damage, heart and blood vessel disease are some complications

    Insulin

    • A hormone produced by the pancreas, crucial for glucose uptake into cells
    • Composed of an A-chain and B-chain linked by disulfide bonds
    • Slightly different structures between species
    • Prior to recombinant technology, porcine insulin was widely used or tested with
    • First peptide hormone discovered and the first protein to be chemically synthesized and produced using DNA recombinant technology

    Insulin: Storage and Form

    • Stored as a hexamer (6 molecules linked together) for stability during storage
    • The active form is the monomer
    • Zinc atoms and water molecules are crucial in keeping the hexamer stable, yet readily available for use

    Insulin: Therapeutic Applications

    • Insulin injections are used to manage high blood glucose levels in diabetes
    • Recombinant human insulin, produced in E. coli, is now common
    • Different types offer varying onset, peak, and duration of action (rapid-acting, intermediate, long-acting).
    • Many newer formulations also exist to improve patient compliance and treatment effectiveness

    Types of Recombinant Insulin

    • Rapid-acting insulin: Used in rapid-acting insulin preparations, faster onset of action, higher maximum plasma concentration (Cmax), shorter duration
    • Intermediate and long-acting insulin: Delayed absorption profile, frequently used to control glucose levels throughout the day, slow dissolving, and remaining near the injection site.
    • Insulin is aerosolized, injected into the airways, degrades DNA to decrease mucus viscosity and promoting secretion clearance.

    Insulin Glargine (insulin glargine analogue):

    • Modified form, one amino acid changed (asparagine replaced by glycine in A-chain), adding two arginines to the end of the B-chain
    • Difference in isoelectric point (pH 7) vs. native insulin (pH 5.4)
    • Leads to its solubility in acidic solution, which promotes slow sustained release when injected
    • Forms microprecipitates after injection from which small amounts of insulin are continuously released to the circulation

    Insulin Absorption

    • Insulin monomers and dimers are readily absorbed by blood capillaries
    • Insulin hexamers are not absorbed/absorbed very slowly
    • Excipients (zinc, phenol-like) added to stabilize hexamers and act as preservatives
    • Lipophilic excipients disperse away from site, allowing dissociation to monomers and dimers to facilitate absorption

    Insulin Therapy

    • Various insulin preparations (rapid-acting, intermediate, long-acting, glargine)
    • Insulin is used to effectively manage blood glucose concentrations, reducing complications and risks
    • Source of insulin: could previously be extracted from pigs and cows; now mostly genetically engineered or produced directly by microorganisms.

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    Recombinant Therapeutic Proteins I PDF 1120-111 1966

    Description

    This quiz tests your knowledge of the role of rhDNase I in cystic fibrosis therapy. It covers various aspects including its mechanisms, biological activity, and the implications of CFTR mutations. Evaluate your understanding of the therapeutic effects and the relationship between rhDNase I and lung function enhancement.

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