Key result
Viral vector-mediated antisense therapy offers a strategy to overcome limitations of antisense oligonucleotides by shuttling sequences into cells for permanent correction of genetic diseases.
Viral vectors encoding small nuclear RNAs offer a promising strategy to overcome the limitations of traditional antisense oligonucleotides by enabling appropriate subcellular localization and permanent correction in genetic diseases.
Antisense oligonucleotides may advance RNA-targeted cardiovascular therapies; leaves open optimal targets, delivery, and clinical validation.
RNA plays complex roles in normal health and disease and is becoming an important target for therapeutic intervention; accordingly, therapeutic strategies that modulate RNA function have gained great interest over the past decade. Antisense oligonucleotides (AOs) are perhaps the most promising strategy to modulate RNA expression through a variety of post binding events such as gene silencing through degradative or non-degradative mechanisms, or splicing modulation which has recently demonstrated promising results. However, AO technology still faces issues like poor cellular-uptake, low efficacy in target tissues and relatively rapid clearance from the circulation which means repeated injections are essential to complete therapeutic efficacy. To overcome these limitations, viral vectors encoding small nuclear RNAs have been engineered to shuttle antisense sequences into cells, allowing appropriate subcellular localization with pre-mRNAs and permanent correction. In this review, we outline the different strategies for antisense therapy mediated by viral vectors and provide examples of each approach. We also address the advantages and limitations of viral vector use, with an emphasis on their clinical application.
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Imbert et al. (2017) conducted a review in Genetic Diseases. Viral Vector-Mediated Antisense Therapy was evaluated. Viral vector-mediated antisense therapy offers a strategy to overcome limitations of antisense oligonucleotides by shuttling sequences into cells for permanent correction of genetic diseases.
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