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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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In-vitro Mutagenesis01:16

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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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Viral Vectors in Gene Replacement Therapy.

Ekaterina Minskaia1, Alima Galieva2, Alexander D Egorov2

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Gene therapy offers long-lasting treatments for rare genetic disorders, moving beyond symptom management. Advances in viral vectors have enabled successful clinical applications across various diseases, providing hope for patients.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Historically, rare genetic disorders were managed solely through symptom relief.
  • Significant advancements in gene therapy research have yielded effective and safe treatments.
  • Improved viral vectors enhance gene delivery, immune evasion, and therapeutic gene expression.

Purpose of the Study:

  • To review the progress and impact of gene therapy for rare genetic disorders.
  • To focus on adeno-associated viruses (AAVs), adenoviruses (AdVs), and lentiviruses (LVs) as key vector platforms.
  • To highlight successful preclinical and clinical gene therapy studies and approved treatments.

Main Methods:

  • Review of scientific literature focusing on gene therapy vector platforms (AAV, AdV, LV).
  • Analysis of preclinical and clinical studies demonstrating efficacy and safety.
  • Examination of approved gene therapies and their target diseases.

Main Results:

  • Successful gene therapies approved for hematological (hemophilia A/B, β-thalassemia), ophthalmological (retinal dystrophy), neurological (spinal muscular atrophy, AADC deficiency, ALD), and metabolic disorders.
  • Six AAV-based therapies (Glybera, Luxturna, Zolgensma, Upstaza, Roctavian, Hemgenix) and three LV-based therapies (Libmeldy, Zynteglo, Skysona) have been approved.
  • Gene therapy has shown significant success in restoring function and treating previously intractable conditions.

Conclusions:

  • Gene therapy represents a paradigm shift from symptom management to potentially curative treatments for rare genetic diseases.
  • Despite challenges in development, approved gene therapies offer substantial hope and improved outcomes for patients.
  • Continued research into vector platforms like AAVs, AdVs, and LVs is crucial for expanding therapeutic options.