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Therapeutic Application of Enzymes

Learning Goals

  • Understand that a deficiency or reduced activity of enzymes can cause disease (e.g., GSD Type II, hyperlipidaemia).
  • Understand how enzymes can be used as therapeutic agents, either directly or in gene therapy-based procedures.
  • Understand how enzymes can be used in many detection methods (i.e., bioluminescence, chemiluminescence, Western blot) and also in a variety of industrial/commercial/pharmaceutical processes (e.g., drug manufacturing).
  • Understand that enzymes can be engineered to improve their efficiency.

Discussion Questions

  • Q: What is the protein component of an enzyme called?
    • A. Holoenzyme
    • B. Coenzyme
    • C. Apoenzyme
    • D. Cofactor
  • Q: What is a coenzyme?
    • A. Protein that helps an enzyme function correctly
    • B. A secondary enzyme that helps a primary enzyme to work
    • C. Non-protein that non-covalently binds to an enzyme to activate it
    • D. Enzyme that inhibits another enzyme

Enzyme Deficiency and Disease

  • Deficiency or reduced activity of enzymes can cause disease.
  • Glycogen Storage Diseases (GSD)
    • Metabolic disorder involving enzymes regulating glycogen metabolism.
    • GSD Type II (Pompe’s disease) caused by deficiency of lysosomal α-glucosidase (accumulation of glycogen in lysosome).
    • Muscle weakness and nerve damage leading to heart failure.
    • Administer myozyme (replaces defective enzyme).

Enzymes as Therapeutic Agents

  • Enzymes can be used as therapeutic agents, e.g., Stroke.
    • Apply an exogenous enzyme to treat the disease process.
    • e.g., Stroke - break up blood clots (streptokinase, tissue plasminogen activator [t-PA], urokinase).
  • Cancer
    • Acute leukaemia cells can have an enzyme deficiency where they are unable to synthesize asparagine.
    • Obtain Asn from healthy tissue as tumour cell growth needs asparagine.
    • Asparaginase limits tumour growth by removing Asn (healthy tissue) available to tumour cells.
      • Asparagine = tumour growth
      • Asparaginase = removes asparagine = cell death
  • Skin Ulcers
    • Break in the skin → underlying tissues can be seen.
    • Severe ulcers can have an accumulation of dead tissue which impairs healing.
    • Topical application of ointment containing collagenase can be used to promote healing.
  • Lipoprotein Lipase Deficiency (Hyperlipidaemia)
    • Defect in lipoprotein lipase.
    • Cannot digest fat from diet.
    • Elevated chylomicrons [transport lipids from the digestive tract to the liver].
    • Pancreatitis, enlargement of liver and spleen, xanthomas (yellow skin lesions).
    • Treatment: limit fat intake and gene therapy.

Gene Therapy

  • Direct Treatment
    • Replace defective gene with normal gene directly into human.
    • Package into virus (use a “no symptoms” virus).
    • Glybera approved for the treatment of lipoprotein lipase deficiency – others that are coming onto the market include valoctocogene roxaparvovec (haemophilia) targets FactorVIII (zymogen).
    • Concerns:
      • Insert in the wrong place?
      • Not all cells get infected (receptors).
      • Protein overload can cause death.
  • Ex vivo Treatment
    • Take out patient cells - place gene into cells and then put cells back into patient.
    • The first successful gene therapy occurred in 1990.
      • Adenosine deaminase deficiency.
      • Purine metabolism (nucleic acid breakdown).
      • Development/maintenance of the immune system.
      • Severe combined immunodeficiency - have no immune system.
      • Gene placed into T lymphocytes.
    • The first patient is still healthy.

Enzymes in Detection Methods

  • Bioluminescence
    • Bioluminescence - release of light.
    • Firefly luciferase (found inside abdomen) transforms luciferin to oxyluciferin + light.
    • Used to detect reactions that otherwise couldn’t be visualised (e.g., tumour cells in animals).
  • Chemiluminescence
    • Chemiluminescence → light produced when a chemical reaction is triggered in the presence of a catalyst.
    • Luminol.
    • Biochemistry: Used to detect proteins, nucleic acids.
    • Clinical: Detection of blood.
    • The presence of heme-enzyme can also cause the release of light (presence of oxidants and basic compounds).
  • Immunochemical Methods
    • Immunochemical - using antibodies (e.g., Western blot).
    • Detects the presence of proteins within a sample allows quantitation.
    • Antibody has enzyme conjugated to it.
    • The enzyme converts substrate to product and colour or light is released.
    • Uses antibodies – perform this in Cell Signalling practical.

Commercial Applications (Food)

  • Many are ‘engineered’ or designed for commercial/industrial applications.
  • Food Processing:
    • e.g., Glucose oxidase = preservation.
    • Rennet from calf stomach = coagulates milk in cheese production.
    • Proteases (e.g., trypsin, pepsin, etc.) = meat tenderizers, prevention of cloudiness in beer.
  • Taste Improvement:
    • e.g., Glucose isomerase = increases the sweetness of drinks.
    • Naringinase = removal of bitter taste in grapefruit juice.
    • Lipase from the pancreas = improvement of cheese aroma.

Industrial Applications

  • Paper and Textile Industries
    • e.g., Glucose isomerase = added to paper to increase its plasticity.
    • Amylase = removal of starch from textiles.
    • Proteases (trypsin, pepsin) = tanning agent for leather.
  • Cosmetics:
    • e.g., Proteases = removal of necrotic tissue, acne treatment.

Pharmaceutical Applications

  • Drug Manufacturing
    • Removal of impurities e.g., removal of contaminating proteins in Phyollophorus proteus using papain.
    • Drug synthesis e.g., synthesis of simvastatin (acyltransferase – LovD).
    • Enantioselective reactions e.g., production of S-citalopram by lipase.
    • Engineered to improve usefulness.

Enzyme Engineering

  • Enzymes can be engineered to improve their activity or efficiency.
  • Recombinant DNA technology → alter amino acid sequence.
  • Alter kinetic properties, regulation, or enhance substrate specificity/activity, increase stability (e.g., heat, solvent, degradation).
  • E.g., Penicillin acylase (used to produce the first reagent in penicillin manufacture) → engineered to improve substrate selectivity.

Discussion Questions

  • Q: What is bioluminescence?
    • A. Metabolism of substrate (luciferase) by enzyme (luciferin) to release light
    • B. Metabolism of substrate (oxyluciferin) by enzyme (luciferase) to release light
    • C. Metabolism of substrate (luciferin) by enzyme (luciferase) to release light
    • D. Metabolism of substrate (luciferase) by enzyme (oxyluciferin) to release light
  • Q: Which of the following modifications of an enzyme would NOT improve the activity or efficiency of the enzyme?
    • A. Alter kinetic properties
    • B. Decrease substrate specificity
    • C. Increase heat stability
    • D. Decrease enzyme degradation

Summary

  • Deficiency or reduced activity of enzymes can cause disease.
  • Enzymes can be used as therapeutic agents e.g., myozyme for GSD Type II, t-PA in stroke, asparaginase in leukaemia, collagenase for skin ulcers.
  • Gene therapy - direct and ex vivo.
  • Enzymes can be used in detection methods e.g., bioluminescence, chemiluminescence, Western blot.
  • Variety of commercial, industrial and pharmaceutical applications for enzymes e.g., food processing, taste improvement, paper/textile industries, cosmetics, drug synthesis.
  • Enzymes can be engineered to improve their activity or efficiency.

Further Reading

  • Alberts et al. Molecular Biology of the Cell. Sixth Edition. Chapters 2 and 3
  • Berg et al. Biochemistry. Sixth Edition. Chapter 8.