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Gene therapy with transcription factor decoy oligonucleotides as a potential treatment for cardiovascular diseases
Naruya Tomita1, Haruhito Azuma, Yasufumi Kaneda
1Department of Geriatric Medicine, Osaka University Graduate School of Medicine, Suita 565-0871, Japan. tomita@hp-gm.med.osaka-u.ac.jp
Abstract:
Cardiovascular diseases including renal diseases are the leading causes of mortality and morbidity in developed countries. Most conventional therapy is inefficient and tends to treat the symptoms rather than the underlying causes of the disorder. Gene therapy based on oligonucleotides (ODN) offers a novel approach for the prevention and treatment of cardiovascular diseases. Gene transfer into somatic cells to interfere with the pathogenesis contributing to cardiovascular disease may provide such a novel approach for better prevention and treatment of cardiovascular disorders. The major development of gene transfer has importantly contributed to intense investigation of the potential of gene therapy in cardiovascular including renal medicine. The amazing advances in molecular biology have provided a dramatic improvement of the technology that is necessary to transfer target genes into somatic cells. Gene transfer methods have been surprisingly improved. In fact, some of them (retroviral vectors, adenoviral vectors or liposome based vectors, etc) have been used in the clinical trials already. Recent progress in molecular biology has provided new techniques to inhibit target gene expression. Especially, application of DNA technology such as an antisense strategy to regulate the transcription of disease-related genes in vivo has important therapeutic potential. Recently, transfection of cis-clement double-stranded ODN (= decoy) has been reported as a new powerful tool in a new class of anti-gene strategies for gene therapy. Transfection of double-stranded ODN corresponding to the cis sequence will result in attenuation of the authentic cis-trans interaction, leading to removal of trans-factors from the endogenous cis-elements with subsequent modulation of gene expression.
Insights
Gene therapy using oligonucleotides (ODN) presents a novel approach to treat cardiovascular and renal diseases by targeting underlying causes. This method modulates gene expression, offering a promising alternative to conventional symptom-focused treatments.
Area of Science:
- Molecular Biology
- Cardiovascular Medicine
- Gene Therapy
Background:
- Cardiovascular and renal diseases are leading causes of mortality globally.
- Conventional therapies often manage symptoms rather than underlying causes.
- Gene therapy offers a novel therapeutic avenue.
Purpose of the Study:
- To explore gene therapy using oligonucleotides (ODN) for cardiovascular and renal diseases.
- To investigate the potential of gene transfer in modulating disease pathogenesis.
- To highlight advances in gene transfer technology and gene expression inhibition.
Main Methods:
- Utilizing gene transfer into somatic cells to interfere with disease mechanisms.
- Employing DNA technology, including antisense strategies, to regulate gene transcription.
- Applying double-stranded ODN (decoy) transfections as an anti-gene strategy.
Main Results:
- Gene transfer methods have significantly advanced, with some used in clinical trials.
- New techniques allow for the inhibition of target gene expression in vivo.
- Double-stranded ODN decoys can modulate gene expression by interfering with cis-trans interactions.
Conclusions:
- Oligonucleotide-based gene therapy shows significant therapeutic potential for cardiovascular and renal disorders.
- Advances in molecular biology enhance the feasibility and efficacy of gene therapy.
- Decoy ODN represent a powerful new tool for modulating gene expression in therapeutic contexts.