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Gene therapy for atherosclerosis

D J Rader1

  • 1Department of Medicine, University of Pennsylvania Health System, Philadelphia 19104-6100, USA.

International Journal of Clinical & Laboratory Research
|January 1, 1997
PubMed
Summary
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New gene therapy strategies show promise for treating atherosclerosis, a systemic cardiovascular disease. While gene replacement is established for specific genetic disorders, protein overexpression offers broader potential for inhibiting disease progression.

Area of Science:

  • Cardiovascular Medicine
  • Genetics
  • Molecular Biology

Background:

  • Atherosclerotic cardiovascular disease remains a significant health concern despite advances in prevention and treatment.
  • Atherosclerosis is a systemic condition, making it a suitable target for systemic gene therapy approaches.
  • Current therapeutic strategies necessitate the development of novel approaches to inhibit disease progression.

Purpose of the Study:

  • To explore the potential of somatic gene transfer as a therapeutic strategy for atherosclerosis.
  • To differentiate systemic gene therapy from localized vascular gene delivery for atherosclerotic lesions.
  • To outline two primary strategies for systemic gene therapy in atherosclerosis: gene replacement and protein overexpression.

Main Methods:

Related Experiment Videos

  • Review of existing literature and clinical trials in gene therapy for cardiovascular diseases.
  • Conceptual framework for systemic gene therapy targeting atherosclerosis.
  • Identification of potential targets and challenges for gene therapy application.

    • Main Results:

    • Gene replacement therapy is feasible for genetic disorders causing premature atherosclerosis, with a pilot trial for homozygous familial hypercholesterolemia reported.
    • Protein overexpression strategies hold potential for broad application in patients at risk for atherosclerosis, independent of etiology.
    • Systemic gene therapy requires further advancements in vector development and validation in animal models.

    Conclusions:

    • Systemic gene therapy presents a promising avenue for treating atherosclerosis by modulating systemic factors.
    • Further research and development, particularly in vector technology and preclinical efficacy, are crucial for clinical translation.
    • Gene therapy offers a potential future therapeutic modality for a large patient population at risk for progressive atherosclerosis.