explain the advantages of using recombinant human proteins to treat disease, using the examples insulin, factor VIII and adenosine deaminase

Genetic Technology Applied to Medicine

Objective

Explain the advantages of using recombinant human proteins to treat disease, using the examples insulin, factor VIII and adenosine deaminase.

Why Use Recombinant Human Proteins?

• Exact human sequence → reduced immunogenicity.
• Consistent supply – not dependent on animal or human donors.
• Scalable production in microbial or mammalian cell culture.
• Ability to engineer modifications (e.g., increased half‑life).
• Purity and safety – removal of pathogens.

Key Examples

1. Recombinant Human Insulin (r‑hINS)

Insulin deficiency in Type 1 diabetes is treated by daily injections of insulin. Recombinant insulin is produced in Escherichia coli or yeast.

• Advantages:

  1. Identical to endogenous human insulin – fewer allergic reactions.
  2. Large‑scale fermentation provides an unlimited supply.
  3. Purity levels > 99 % reduce risk of contaminants.
  4. Formulation flexibility (rapid‑acting, long‑acting analogues).

2. Recombinant Human Factor \cdot III (r‑hF \cdot III)

Factor VIII deficiency causes haemophilia A. Traditional therapy used plasma‑derived F \cdot III, which carried infection risks.

• Advantages:

  1. Eliminates transmission of blood‑borne viruses (HIV, hepatitis).
  2. Consistent activity units – precise dosing.
  3. Reduced batch‑to‑batch variability.
  4. Potential for bio‑engineering longer‑acting products.

3. Recombinant Human Adenosine Deaminase (r‑ADA)

Severe combined immunodeficiency (SCID) due to ADA deficiency is treated with enzyme replacement therapy.

• Advantages:

  1. Provides the exact human enzyme, restoring metabolic balance.
  2. Avoids the need for bone‑marrow transplantation.
  3. Can be produced in Chinese hamster ovary (CHO) cells with proper glycosylation.
  4. Improves patient survival and quality of life.

Comparative Summary

Key Points to Remember

1. Recombinant technology enables production of proteins that are chemically identical to their natural human counterparts.
2. Safety is markedly improved by eliminating reliance on human or animal donors.
3. Scalability ensures that life‑saving medicines are available to a growing patient population.
4. Engineering possibilities open the way for next‑generation therapeutics with enhanced pharmacokinetics.