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希望、绝望、CRISPR:拯救一个女孩生命的竞赛_Uditi ...
Ghosh与他的同事们正在紧急设计一种独特的疗法,意在编辑这位20岁女性大脑细胞中的DNA,让它们停止产生有毒蛋白。 这是一种前所未试的方法,背后有诸多可能失败的原因。 Ghosh致力于制备AAV,该AAV将用于将CRISPR成分输送到Uditi的神经元中。
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