Genetics · MU Sofia

65. Gene therapy - principles, possibilities, applications.

Explains gene therapy as a strategy that modifies a patient's genetic material to treat or prevent disease, by adding a functional gene, correcting a defective one or silencing harmful expression. It contrasts somatic with germline approaches, compares viral vectors (retroviruses, lentiviruses, adenoviruses, AAV) with non-viral ones, and outlines ex vivo and in vivo delivery. It then covers gene editing steps, monogenic, cancer and infectious-disease applications, limitations, ethical issues and future directions.

Inside: 13 sections · 12 labelled pictures · 15 flashcards · 5 min

Topic 65 of 65 in the MU Sofia syllabus for Genetics (Medicine, Year 3). The free AceMed note covers it in 13 sections, about 599 words (5 min of reading), with 15 flashcards.

In this note

  1. Gene therapy - principles, possibilities, applications.
  2. I. DEFINITION AND GENERAL CONCEPT
  3. II. BASIC PRINCIPLES OF GENE THERAPY
  4. III. TYPES OF GENE THERAPY
  5. IV. GENE DELIVERY SYSTEMS (VECTORS)
  6. V. METHODS OF GENE THERAPY APPLICATION
  7. VI. GENE EDITING TECHNOLOGIES
  8. VII. APPLICATIONS OF GENE THERAPY
  9. B. CANCER GENE THERAPY
  10. C. GENE THERAPY FOR INFECTIOUS AND ACQUIRED DISEASES
  11. VIII. LIMITATIONS AND RISKS OF GENE THERAPY
  12. IX. ETHICAL AND SOCIAL CONSIDERATIONS
  13. X. FUTURE POSSIBILITIES OF GENE THERAPY

I. DEFINITION AND GENERAL CONCEPT

Gene therapy is a therapeutic strategy that aims to treat or prevent disease by modifying genetic material in a patient’s cells.

This can be achieved by:

  • introducing a functional gene,
  • correcting a defective gene,
  • silencing or modifying gene expression.

Unlike conventional therapy, gene therapy attempts to address the root genetic cause of disease rather than only treating symptoms.

Key Concept

Gene therapy targets the genotype in order to correct the phenotype, making it fundamentally different from enzyme replacement or symptomatic treatment.

II. BASIC PRINCIPLES OF GENE THERAPY

For gene therapy to be effective:

  • the target gene must be well characterized,
  • the disease mechanism must be understood,
  • an appropriate delivery system (vector) must be available,
  • gene expression must be sufficient and regulated,
  • adverse immune responses must be minimized.

Main Strategies of Gene Therapy

1. Gene Addition

A functional copy of a gene is introduced to compensate for a defective one.

This is the most commonly used approach.

2. Gene Correction

The mutant gene is directly repaired or edited to restore normal function.

3. Gene Silencing

Expression of a harmful gene is reduced or blocked (e.g. dominant-negative mutations).

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