現今已知接近有7萬幾種的基因變異會引起疾病。417Please respect copyright.PENANAgITE2ZDLmb417Please respect copyright.PENANAIScEC6PEps
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隨著CRISPR基因編輯技術的興起,將會為根治這些疾病帶來曙光。417Please respect copyright.PENANAvLq6JJwA7I
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(圖2)圖中將展示「舊式」的編輯方法HDR(Homology Directed Repair)機制。417Please respect copyright.PENANAIyJUCLl3U2417Please respect copyright.PENANAj5pERZ8Igd
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sgRNA將會引領Cas9蛋白質抵達不理想的基因突變位置,其後Cas9蛋白質會將基因斬斷成兩截;417Please respect copyright.PENANAzJHEqVTVn6
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根據Cas9蛋白質的特性,可修改至只斬一條特定的DNA位置,其後會再提及。417Please respect copyright.PENANAo5XkAWRJHr
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(圖3)此時候,如果將盛載想修改的基因排組送到斷成兩截的基因附近,細胞便會加以利用,將原本的紫色區域改成為黃色區域。417Please respect copyright.PENANAutJAR5Gotq417Please respect copyright.PENANA48rvSFr4PY
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但這個方法的隨機性非常之高,最終結果有可能會出現基因刪減,又或者是其他基因排組的插入。417Please respect copyright.PENANAcDFupqYVoV
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套用基因編輯技術於治療病人身上的話,精準度必須要非常之高,此手段明顯並不理想。417Please respect copyright.PENANAmIKswvVhvo
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(圖4)至於新式編輯方法Prime Editing方面,同樣亦是套用CRISPR技術,但各組件及理念則有所修改。417Please respect copyright.PENANAicrnFh5a0R417Please respect copyright.PENANAYEziYOVCmc
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擁有Transcriptase Domain的Cas9變種蛋白質將會被含有預想修改基因排組的pegRNA引領到不理想的基因突變位置,而這一次只會斬斷特定的一條DNA。417Please respect copyright.PENANAhhH3GeDYoT
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(圖5)其後,Cas9變種蛋白質會讀取pegRNA內含的預想修改基因排組,並將一條DNA的基因排組成功修改。417Please respect copyright.PENANAqt3BkBDCET417Please respect copyright.PENANAzOCM16zLOS
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經過一系列難以深入淺出的程序後,最終亦會完成成功修改雙方兩截的DNA。417Please respect copyright.PENANATEePoNQxFZ
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(圖6)紅色區域為不理想的基因修改結果(Ref2),新式編輯方法PrimeEditing明顯優勝於舊式編輯方法HDR,但仍有一定程度的進步空間。417Please respect copyright.PENANA4LOM9SAQEE417Please respect copyright.PENANA6W0u2uphUh
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有朝一日,CRISPR基因編輯技術定必會在治療疾病上得以普及化。417Please respect copyright.PENANAHKmdD6LJ4P
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https://www.penana.com/user/27349/%E8%96%AF%E4%BB%94%E8%81%9E%E8%8C%B1%E8%8E%89/portfolio417Please respect copyright.PENANAOPMKc1k441
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Ref1:https://www.nature.com/articles/s41586-019-1711-4417Please respect copyright.PENANAb9l3qnzPZx
Ref2:https://www.nature.com/articles/s41467-020-19136-7
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