CRISPR基因疗法的实现。
The realization of CRISPR gene therapy.
作者信息
Ma Yitong, Qi Lei S
机构信息
Department of Bioengineering, Stanford University, Stanford, CA, USA.
Sarafan ChEM-H, Stanford University, Stanford, CA, USA.
出版信息
Nat Chem Biol. 2024 Jul;20(7):791-795. doi: 10.1038/s41589-024-01645-x.
Abstract
Casgevy, the inaugural CRISPR-based drug, has been approved by several medical agencies and represents an important milestone in gene therapy. Additional CRISPR-based therapies are currently in clinical trials and could be approved soon. Although there are technological hurdles to overcome, chemical biology has a vital role to play in developing recent breakthroughs in base editing, prime editing, and epigenetic editing into future treatments.
摘要
首款基于CRISPR的药物“凯雪维”（Casgevy）已获多家医学机构批准，是基因治疗领域的一个重要里程碑。目前还有其他基于CRISPR的疗法正在进行临床试验，可能很快会获批。尽管仍有技术障碍有待克服，但化学生物学在将碱基编辑、先导编辑和表观遗传编辑等近期突破转化为未来治疗方法方面将发挥至关重要的作用。
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