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Biochemistry · Enzymology

Enzymes in medicine and enzymopathies

Enzyme therapy is the use of specific enzymes as adjunctive medications for the treatment of various diseases. In contrast, enzymopathies represent pathological conditions that develop due to impaired normal functioning of intracellular enzymes.

Enzyme therapyThe use of enzyme preparations for the treatment of a wide range of pathologies.
HeredityPrimary enzymopathies are always associated with genetic defects.
Blood malignanciesAsparaginase deprives leukemic cells of a necessary amino acid.
SurgeryProteases effectively clear purulent wounds of dead cells.
4 min readFor medical studentsUpdated 09/24/2026
Material prepared by WhiteCoat Pro editorial team based on platform study guidesReviewed by medical editor: Aulov A. A., general practitioner · 09/24/2026

Main areas of enzyme therapy

Enzyme preparations are actively being introduced into clinical practice as adjunctive therapy. Depending on substrate specificity, they are prescribed for inflammation, thrombosis, viral infections, and even oncology.

1. Preparations with proteolytic activity This group includes trypsin and chymotrypsin. They are used exclusively topically. The main task of such enzymes is the cleavage of proteins in necrotic tissues. This makes them indispensable in the management of purulent wounds. In addition, proteases help remove blood clots and effectively liquefy excessively viscous secretions that accumulate during respiratory tract inflammation.

2. Use of nucleases The enzymes ribonuclease and deoxyribonuclease are used in medicine as specific antiviral agents. Their direct indication is the treatment of viral eye lesions, such as adenoviral conjunctivitis and herpetic keratitis.

3. Fibrinolytic agents To combat thrombosis, medications capable of dissolving already formed blood clots are used. This process is called thrombolysis. The physician's arsenal includes drugs such as fibrinolysin, streptokinase (also known as streptolyase), streptodornase [Note: streptokinase derivative / streptodornase], and urokinase. The main indications for their administration are life-threatening thrombosis and thromboembolism.

Specific enzymes: Hyaluronidase and Asparaginase

In addition to proteases and fibrinolytics, enzymes with a narrow mechanism of action are used in therapy.

  • Hyaluronidase (Lidase). This enzyme is a catalyst for the breakdown of hyaluronic acid, an important component of connective tissue. The drug is administered subcutaneously or intramuscularly. The main goal of application is the resolution of pathological scars (e.g., after deep burns) and postoperative adhesions.
  • Asparaginase. This enzyme has found its application in oncohematology, particularly in the treatment of blood disorders (leukemias). Its mechanism of action is based on the destruction of the amino acid asparagine (Asn) directly in the bloodstream. A characteristic feature of leukemic cells is that they have lost the ability to independently synthesize this element and are completely dependent on its influx from the blood. By destroying asparagine, the enzyme deprives tumor cells of building material, leading to growth arrest.
Test your knowledge on this topicTasks from the Biochemistry course bank — instant answer and detailed explanation.

What are enzymopathies?

If enzyme therapy is treatment using enzymes, then enzymopathies are diseases whose root cause is a malfunction of the cell's own enzymes.

Globally, all enzymopathies are divided into two large categories:

  1. Primary. These are hereditary pathologies in which the enzyme defect is determined at the genetic level.
  2. Secondary. Such enzymopathies are acquired. They accompany most known diseases and essentially proceed as proteinopathies (pathologies of protein structures).

Briefly: answers to topical questions

Which specific diseases belong to primary hereditary enzymopathies?

Primary hereditary enzymopathies include diseases classified by the type of metabolic disorder:

  • Amino acid metabolism disorders — phenylketonuria, albinism, alkaptonuria, tyrosinosis.
  • Carbohydrate metabolism disorders — galactosemia, hereditary fructose intolerance, glycogen storage diseases.
  • Lipid metabolism disorders — lipidoses.
  • Nucleic base metabolism disorders — gout, Lesch-Nyhan syndrome.
  • Connective tissue metabolism disorders — mucopolysaccharidoses, chondrodystrophy.
  • Gastrointestinal enzyme defects — cystic fibrosis, celiac disease, lactose intolerance.

Hereditary enzymopathies also include acute intermittent porphyria and desmosis.

Which enzyme preparations are used for replacement therapy in gastrointestinal diseases?

For replacement therapy in gastrointestinal tract diseases, medications that compensate for insufficient secretion of digestive juices are used.

  • Pepsin — used for gastric therapy in gastritis with reduced secretory function.
  • Festal, enzistal, mezim-forte, and pancreatin — used to compensate for the deficiency of major pancreatic enzymes.
  • Microspheric pancreatic enzymes — modern medications used to manage maldigestion syndrome in exocrine insufficiency.

What are the methods and approaches for treating primary enzymopathies?

For the treatment of primary hereditary enzymopathies as diseases associated with enzyme gene defects, the following approaches directly follow from the sources:

  • Symptomatic treatment — elimination of symptoms.
  • Pathogenetic treatment — diet therapy in phenylketonuria, replacement therapy, removal of toxins.
  • Replacement enzyme therapy — the use of enzymes in case of their deficiency in the body.
  • Etiological treatment — gene therapy, considered a promising direction.
How to memorize

To remember the indications for nucleases, associate the root "nucleo-" (nucleus, viral DNA/RNA) with the antiviral effect in eye infections (keratitis, conjunctivitis).

Class and exam questions

How do proteolytic enzymes work in surgery?

Trypsin and chymotrypsin are used topically. They catalyze the breakdown of proteins in dead cells, thereby clearing purulent wounds and dissolving blood clots.

Which drugs belong to fibrinolytics and why are they needed?

These include fibrinolysin, streptokinase, streptodornase, and urokinase. They are essential for thrombus destruction (thrombolysis) in thrombosis and thromboembolism.

How and why is hyaluronidase used?

Hyaluronidase (Lidase) is administered intramuscularly or subcutaneously. It breaks down hyaluronic acid, facilitating the resolution of scars after burns and adhesions after surgeries.

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What else to study on this topic

  • Molecular mechanisms of trypsin and chymotrypsin action
  • Examples of hereditary (primary) enzymopathies
  • Pathogenesis of secondary enzymopathies as proteinopathies
  • Biochemical mechanism of thrombolysis by fibrinolytics
  • Asparagine metabolism in healthy and tumor cells

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