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      2. west china medical publishers
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        find Keyword "CRISPR/Cas9" 2 results
        • The progress of the gene editing therapy of inherited retinal diseases based on CRISPR/Cas9

          Inherited retinal diseases (IRDs) are the major cause of refractory blinding eye diseases, and gene replacement therapy has already made preliminary progress in the treatment of IRDs. For IRDs that cannot be treated by gene replacement therapy, gene editing provides an alternative therapeutic method. Strategies like disruption of pathogenic variants with or without gene augmentation therapy and precise repair of pathogenic variants can be applied for IRDs with various inheritance patterns and pathogenic variants. In animal models of retinitis pigmentosa, Usher syndrome, Leber congenital amaurosis, cone rod cell dystrophy, and other disorders, CRISPR/Cas9, base editing, and prime editing showed the potential to edit pathogenic variations in vivo, indicating a promising future for gene editing therapy of IRDs.

          Release date:2023-08-17 08:49 Export PDF Favorites Scan
        • CRISPR/Cas9 技術在乙型肝炎病毒基因組抑制中的應用

          目前世界范圍內約有 2.4 億慢性乙型肝炎病毒(hepatitis B virus,HBV)感染者,HBV 感染是世界性的重大公共衛生難題。隨著分子生物學工具的不斷發展,目前第 3 代基因定點編輯技術 CRISPR/Cas9 作為熱點已經廣泛地應用于多種病毒的研究與實驗性治療中。該文簡要回顧了 HBV 基因組的特點、基因編輯技術的發展及原理和 CRISPR/Cas9 在 HBV 基因組抑制中的研究現狀及局限性。相對于鋅指核糖核酸酶和轉錄激活因子樣效應物核酸酶其他兩種基因編輯技術,CRISPR/Cas9 技術極大地提高了基因編輯的能力。雖然目前仍屬于概念證明階段,但多數基礎研究均證實了 CRISPR/Cas9 技術在體內外對 HBV 基因組具有編輯能力并能降低其 DNA 復制與病毒蛋白的表達能力。在潛在安全風險及基因編輯載體的輸送效率等問題得到解決后,CRISPR/Cas9 技術聯合逆轉錄抑制藥物的治療將為 HBV 感染的臨床治愈帶來曙光。

          Release date:2017-12-25 06:02 Export PDF Favorites Scan
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          2. 射丝袜