Therapeutic Applications of Enzymes and Isoenzymes

  • Therapeutic Applications of Enzymes and Isoenzymes refer to the use of naturally occurring or engineered enzymes and their isoforms (isoenzymes) in the diagnosis, treatment, and management of diseases.
  • These Therapeutic Applications include enzyme replacement therapies, clot dissolution, anti-inflammatory treatments, cancer therapy, and diagnostic biomarker

Enzyme Replacement Therapy (ERT):

  • Purpose: To compensate for deficient or malfunctioning enzymes in patients with inherited metabolic disorders.
  • Examples:

    • Gaucher Disease: Caused by a deficiency of glucocerebrosidase. ERT with recombinant glucocerebrosidase (imiglucerase) reduces the accumulation of glucocerebroside in tissues.
    • Fabry Disease: Deficiency of α-galactosidase A leads to lipid accumulation. Administration of recombinant α-galactosidase A alleviates symptoms.
    • Pompe Disease: Lack of acid α-glucosidase causes glycogen buildup. ERT with alglucosidase alfa helps reduce glycogen storage in muscles.
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Thrombolytic Therapy:

  • Purpose: To dissolve blood clots in conditions like myocardial infarction, pulmonary embolism, and ischemic stroke.
  • Enzymes Used:

    • Streptokinase: Activates plasminogen to plasmin, which breaks down fibrin clots.
    • Urokinase: Directly converts plasminogen to plasmin.
    • Tissue Plasminogen Activator (tPA): Recombinant enzyme that specifically targets fibrin-bound plasminogen, minimizing systemic effects.

Cancer Therapy:

  • Asparaginase: Used in treating acute lymphoblastic leukemia (ALL). It depletes asparagine, an amino acid essential for the survival of certain leukemia cells, leading to cell death.
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Digestive Aids:

  • Pancreatic Enzymes: Such as pancrelipase, a mixture of lipase, amylase, and protease, used in patients with pancreatic insufficiency to aid digestion.
  • Lactase Supplements: Help individuals with lactose intolerance digest lactose in dairy products.

Anti-Inflammatory Agents:

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Wound Debridement:

  • Collagenase and Papain: Enzymes applied topically to remove dead tissue and promote healing in chronic wounds and burns.

Treatment of Gout:

  • Uricase (Rasburicase): Converts uric acid to allantoin, which is more soluble and easily excreted, reducing uric acid levels in patients with gout or tumor lysis syndrome.

Enzyme Therapy for Genetic Disorders:

  • Adenosine Deaminase (ADA) Deficiency: Treated with PEGylated ADA (pegademase bovine) to restore immune function in patients with severe combined immunodeficiency (SCID).
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