Therapy of genetic diseases: a three-level approach

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Updated: Aug 10, 2026

In Vivo Modeling of the Morbid Human Genome using Danio rerio
12:31

In Vivo Modeling of the Morbid Human Genome using Danio rerio

Published on: August 24, 2013

L E Karp

    Postgraduate Medicine
    |February 1, 1980
    PubMed

    Insights

    Genetic disease treatments target different stages, from symptom management to gene correction. Current research focuses on addressing the root genetic defect for potential cures.

    Area of Science:

    • Genetics
    • Medical Science
    • Pharmacology

    Background:

    • Genetic diseases present complex therapeutic challenges.
    • Treatment strategies vary based on disease progression and available interventions.

    Purpose of the Study:

    • To outline the different levels of therapeutic intervention for genetic diseases.
    • To categorize current and emerging treatment modalities.

    Main Methods:

    • Symptomatic treatment after clinical manifestation (diet, drugs, surgery).
    • Gene product replacement therapy before clinical manifestation (e.g., insulin, factor VIII).
    • Gene defect correction at the disease origin (under investigation).

    Main Results:

    • Three distinct therapeutic levels identified: symptomatic, gene product replacement, and gene correction.
    • Symptomatic therapies manage disease after onset.
    • Gene product replacement addresses deficiencies before symptoms appear.

    Conclusions:

    • Therapeutic approaches for genetic disorders are stratified according to disease evolution.
    • Gene defect correction represents the most advanced and investigational level of treatment.
    • Future therapies aim to correct genetic defects at their origin.

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