The World Ahead 2024
世界展望2024
New medical treatments will use genetic scissors, and other clever tricks
新疗法将使用基因剪刀和其他妙招
From sickle-cell disease to glaucoma, these are the drugs to look out for
从镰状细胞病到青光眼，这些是值得留意的药物
- sickle 镰刀
- glaucoma 青光眼
NEW MEDICINES to treat sickle-cell disease and beta thalassaemia, two genetic blood disorders, will make headlines in 2024. Most notable of these is the first CRISPR-gene-edited drug, which made its historic arrival in late 2023. Gene editing uses molecular scissors to edit DNA. It is a more precise form of modification than gene therapy, an older technology that uses a viral vector to inject a working gene into a cell. Gene editing has moved astonishingly quickly through drug pipelines—much faster than gene therapies, which have been slow and difficult to develop.
治疗镰状细胞病和β地中海贫血这两种遗传性血液疾病的新药将在2024年登上新闻头条。其中最引人注目的是首款CRISPR基因编辑药物，它于2023年底历史性地问世了。基因编辑使用分子剪刀编辑DNA，比起使用病毒载体将治疗基因注入细胞的旧技术基因疗法，这种修饰形式更为精确。用基因编辑制造新药的进展快得惊人——相比研发缓慢又困难的基因疗法要快得多。
- thalassaemia 地中海贫血、地中海贫血病、地中海贫血症
- molecular 分子的、与分子有关的
For sickle-cell disease, the gene-edited therapy, exa-cel, developed by Crispr Therapeutics and Vertex, is likely to be approved just ahead of a gene-therapy drug from Bluebird Bio, lovo-cel. In both cases, stem cells are first extracted from a patient’s body. They are then either edited (exa-cel) or transfected with the viral vector (lovo-cel), and returned to the body, where they correct the genetic defect. The effects are said to last a lifetime.
在镰状细胞病上，由Crispr Therapeutics和Vertex研发的基因编辑药物exa-cel很可能会比Bluebird Bio的基因疗法lovo-cel先一步获批。两种疗法都先从患者体内提取干细胞，然后编辑它们（exa-cel）或用病毒载体转染（lovo-cel），然后回输到患者体内，纠正遗传缺陷。据称疗效持续一生。
- viral 病毒的；病毒性的；病毒引起的
- defect 表示“缺点、缺陷、毛病”，同样意味着缺少或缺乏某种对于事物完整或完美来说至关重要的东西（参见：小词详解 | defect）
