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  • Open Access

    REVIEW

    Spliceosome-mediated RNA trans-splicing: a strategy for Huntington’s disease gene therapy

    QINGYANG ZHANG, SHUXIAN HUANG, DAN WENG*

    BIOCELL, Vol.48, No.10, pp. 1443-1453, 2024, DOI:10.32604/biocell.2024.053794 - 02 October 2024

    Abstract Huntington’s disease (HD) is a debilitating neurodegenerative disorder caused by an abnormal expansion of CAG repeats (Cytosine, Adenine, Guanine) in the huntingtin gene (HTT). This mutation leads to the production of a mutant huntingtin protein, resulting in neuronal dysfunction and cell death. Current treatments primarily focus on symptomatic relief and do not address the underlying genetic cause. This review explores spliceosome-mediated RNA trans-splicing (SMaRT) therapy as an innovative and potential approach for HD treatment. SMaRT leverages the cell’s natural splicing machinery to correct mutant mRNA, thereby reducing toxic protein levels while restoring functional protein production. We More >

  • Open Access

    REVIEW

    Differential mRNA expression in peripheral blood is associated with oral squamous cell carcinoma: Recent advances and future challenges

    XIA MU1,2,#, YUBING HU3,#, DANDAN WU1,#, HONGYU YANG1,2,*

    BIOCELL, Vol.47, No.7, pp. 1449-1458, 2023, DOI:10.32604/biocell.2023.026704 - 21 June 2023

    Abstract Oral squamous cell carcinoma (OSCC) is a malignant tumor triggered by the accumulation of multiple gene mutations in oral epithelial cells. Different OSCC-related biomarkers have been reported in circulation in the peripheral blood that support the occurrence and development of OSCC. Recent advances in high-throughput and highly sensitive detection methods have overcome the limitation of the low concentration of most peripheral blood biomarkers. Hence, blood biomarker detection has become an efficient screening tool for the early diagnosis of OSCC. The growing data available in public cancer and gene databases have provided new foundations for OSCC… More >

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