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miRNA Pathway Alteration in Response to Non-Coding RNA Delivery in Viral Vector-Based Gene Therapy

期刊

出版社

MDPI
DOI: 10.3390/ijms232314954

关键词

miRNA pathways; adeno-associated virus; adenovirus; lentivirus; vector; gene therapy; non-coding RNA

资金

  1. Ministry of Science and Higher Education of the Russian Federation
  2. [075-15-2019-1665]

向作者/读者索取更多资源

Gene therapy is commonly used for treating incurable disorders, often using viral vectors to deliver genetic material. However, viral vectors have disadvantages such as immune response and disruption of cell metabolism, which require evaluation of side effects and safety. Many gene therapy techniques use non-coding RNA to regulate gene expression, but this can affect cellular miRNA pathways.
Gene therapy is widely used to treat incurable disorders and has become a routine procedure in clinical practice. Since viruses can exhibit specific tropisms, effectively penetrate the cell, and are easy to use, most gene therapy approaches are based on viral delivery of genetic material. However, viral vectors have some disadvantages, such as immune response and cytotoxicity induced by a disturbance of cell metabolism, including miRNA pathways that are an important part of transcription regulation. Therefore, any viral-based gene therapy approach involves the evaluation of side effects and safety. It is possible for such effects to be caused either by the viral vectors themselves or by the delivered genetic material. Many gene therapy techniques use non-coding RNA delivery as an effective agent for gene expression regulation, with the risk of cellular miRNA pathways being affected due to the nature of the non-coding RNAs. This review describes the effect of viral vector entry and non-coding RNA delivery by these vectors on miRNA signaling pathways.

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