L2. Recombinant Therapeutic Proteins I - AI MCQs
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Questions and Answers

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. (D)</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. (D)</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. (D)</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. (D)</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. (A)</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. (A)</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. (B)</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. (A)</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. (B)</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. (A)</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. (B)</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. (A)</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. (B)</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. (A)</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. (C)</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. (C)</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. (C)</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. (C)</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. (C)</p> Signup and view all the answers

Which statement about recombinant therapeutic proteins is correct?

<p>They can be used to address genetic disorders. (A)</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. (A)</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. (B)</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. (A)</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. (B)</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. (C)</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. (D)</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. (B)</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. (D)</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. (A)</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. (D)</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. (C)</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. (D)</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. (A)</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. (D)</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. (C)</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. (B)</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. (C)</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. (D)</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. (D)</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. (A)</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. (A)</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. (C)</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. (B)</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. (D)</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. (C)</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. (B)</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. (D)</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. (B)</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. (C)</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. (B)</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. (B)</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. (A)</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. (B)</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. (B)</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. (A)</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. (C)</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. (B)</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. (C)</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. (D)</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. (D)</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. (D)</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. (D)</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. (A)</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. (D)</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. (A)</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. (B)</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. (B)</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. (D)</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. (D)</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. (B)</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. (C)</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. (D)</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. (D)</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. (C)</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. (A)</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. (C)</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. (A)</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. (A)</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. (D)</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. (A)</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. (B)</p> Signup and view all the answers

Flashcards

CFTR protein function

Regulates chloride (Cl-) permeability in exocrine glands, maintaining salt and water balance.

Cystic Fibrosis Cause

Mutation in the gene for the CFTR protein

CFTR protein deficiency effect - Cl-

Chloride (Cl-) secretion is blocked in the cells.

CFTR protein deficiency effect- Na+

Sodium (Na+) and water remain in the cells.

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Viscous secretions in CF

Thick, sticky mucus builds up due to inadequate chloride secretion.

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Effects of CF- Lungs

Thick mucus accumulation in the lungs.

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Effects of CF - Pancreas

Thick secretions also affect the pancreas.

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CF Cause: Missing Phenylalanine

A specific mutation that causes the loss of a phenylalanine in the CFTR protein, disrupting its structure/function.

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Recombinant therapeutic proteins

Proteins produced using genetic engineering techniques to treat diseases.

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Cystic fibrosis

A genetic disease affecting lung function, caused by a defective gene.

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rhDNase

A recombinant protein used to treat cystic fibrosis by thinning mucus.

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Type 1 diabetes mellitus

A disease where the body doesn't produce insulin.

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Insulin

A hormone regulating blood sugar levels.

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Recombinant Insulin

Insulin produced using genetic engineering techniques.

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Genetic factors

Inherited traits that influence disease risk.

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Disease pathways

Processes in the body that lead to disease.

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Therapeutic applications

How a treatment is used to manage or cure a disease.

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Diabetes management

Methods and treatments to control diabetes.

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What is rhDNase I?

A recombinant human deoxyribonuclease I (enzyme) produced in mammalian cells, specifically designed for therapeutic use.

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What's rhDNase I's role in CF?

It reduces the viscosity of thick mucus in the lungs of cystic fibrosis patients by breaking down DNA within the mucus.

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How does rhDNase I improve lung function?

By reducing mucus viscosity, it allows better clearance of mucus from airways, improving respiratory function and reducing bacterial infections.

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What is the mechanism of rhDNase I action?

It breaks down DNA in the mucus into smaller fragments, decreasing the mucus's viscoelasticity.

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What is the impact of rhDNase I on DNA

It reduces the size of DNA in mucus by breaking it down into smaller fragments.

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Impaired mucociliary clearance

Abnormal CFTR activity results in poor movement of mucus and cilia in the airways.

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Chronic bacterial infection

A continuous bacterial infection in the respiratory system caused by the impaired CFTR protein.

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Neutrophil-dominated inflammation

Inflammation in the airways is largely caused by an excess of neutrophils, leading to lung damage.

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Viscosity of mucus

The thickness of mucus in the lungs increases due to dead neutrophils and DNA buildup.

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Lung Damage

Repeated infections, blocked airways, and neutrophil damage lead to long-term lung tissue harm.

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Cystic Fibrosis lung function decline

Lung function worsens due to the ongoing issues with infections, damage and mucus, hindering everyday activities.

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Pancreatic inflammation

Inflammation within the pancreas can occur alongside issues with the respiratory system.

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Vitamin deficiencies

Reduced absorption of fat-soluble vitamins occurs due to issues with the pancreas and the digestive system.

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Thick mucus

Thick mucus prevents normal function of the lungs and pancreas. This is due to genetic problems and inflammation.

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Recombinant Protein

A protein artificially produced using genetic engineering techniques. It's made by inserting a human gene into a bacterium, which then produces the desired protein.

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Cystic Fibrosis (CF)

A genetic condition caused by a faulty gene affecting salt and water movement within cells. This leads to thick, sticky mucus build-up in the body's tubes and passages, especially lungs and digestive system.

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What causes Cystic Fibrosis?

A faulty gene affects the movement of salt and water in and out of cells, leading to thick mucus build-up. This faulty gene is responsible for creating the CFTR protein.

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CFTR Protein

A protein that regulates salt and water balance within cells. A faulty CFTR protein causes Cystic Fibrosis.

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Why does thick mucus build up in CF?

The faulty CFTR protein disrupts salt and water movement, leading to a build-up of thick, sticky mucus in the lungs and digestive system.

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Impact of CF on Lungs

Thick mucus builds up in the lungs, making it difficult to breathe and increasing the risk of infections.

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Impact of CF on Digestive System

Thick mucus builds up in the digestive system, interfering with digestion and absorption of nutrients.

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How many recombinant proteins are FDA approved?

Over 130 recombinant proteins are approved by the US FDA for clinical use, showcasing the growth and success of this technology.

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What is the role of recombinant proteins in medicine?

Recombinant proteins are used in research and as drugs for treating various diseases. They offer a targeted and effective approach to therapy.

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How does rhDNase I work?

rhDNase I breaks down DNA in thick CF mucus, reducing its viscosity and allowing easier expectoration.

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What's the benefit of rhDNase I in CF treatment?

It improves lung function by reducing mucus viscosity, allowing better expectoration and reducing infection risk.

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Why is rhDNase I aerosolized?

It is delivered directly into the airways where it acts on thick mucus to improve its clearance.

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When was rhDNase I approved for CF treatment?

It was approved by the FDA in 1993, making it a major breakthrough in CF care.

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Cystic Fibrosis: Effect on Lungs

Thick, sticky mucus builds up in the lungs due to impaired chloride ion (Cl-) transport, leading to difficulty breathing and increased risk of infections.

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Cystic Fibrosis: Effect on Pancreas

Thick mucus also affects the pancreas, interfering with the production and release of digestive enzymes, leading to difficulty digesting food and absorbing nutrients.

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How do recombinant proteins work?

These engineered proteins are designed to target specific disease pathways, sometimes by replacing a faulty protein or by interfering with a disease process.

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What is the impact of rhDNase in CF?

It improves lung function by breaking down DNA in the thick mucus of CF patients, making it easier to clear and reducing bacterial infections.

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Benefits of Recombinant Insulin

It provides patients with type 1 diabetes a way to manage their blood sugar levels and live longer, healthier lives.

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How does genetic engineering help treat diseases?

It allows us to create proteins, like rhDNase and recombinant insulin, that replace missing or faulty proteins, addressing the root cause of genetic diseases.

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What are disease pathways?

These are the processes within the body that contribute to the development of a disease.

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Therapeutic applications of recombinant proteins

They are used to treat a variety of diseases by replacing missing or faulty proteins, interfering with disease pathways, or delivering specific therapies.

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CFTR Mutation

A change in the CFTR gene's DNA sequence that leads to a faulty or missing CFTR protein.

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Thick Mucus in CF

Sticky, thick mucus builds up in the lungs and digestive system due to the defective CFTR protein.

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Lung Damage in CF

Thick mucus traps bacteria, causing chronic infections and inflammation, which damages lung tissue.

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Pancreatic Inflammation in CF

The pancreas can become inflamed due to thick mucus blocking its ducts, affecting digestion and nutrient absorption.

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Vitamin Deficiencies in CF

The body struggles to absorb fat-soluble vitamins because of pancreatic issues, leading to deficiencies.

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Chronic Bacterial Infections in CF

Thick mucus in the lungs provides a breeding ground for bacteria, leading to frequent and persistent infections.

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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

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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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