Philip R Jansen, Mieke van Haelst, Rob M F Wolthuis
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引用次数: 0
摘要
CRISPR-Cas 技术是一种修改 DNA 序列的革命性技术。由于其有效性和准确性,CRISPR-Cas 为各种遗传性疾病的 "基因治疗 "带来了重要希望。CRISPR-Cas 使研究人员能够以前所未有的精度修改基因组的特定部分,在特定情况下,可用于纠正致病 DNA 变异。然而,一些重要的障碍使临床应用的轨迹变得复杂。例如,人们对其安全性、有效性和伦理合理性存在担忧。尽管如此,随着 CRISPR-Cas 被越来越多的人所熟知,医生和医疗服务提供者应该充分了解 CRISPR-Cas 疗法的发展情况。专业知识以及与遗传疾病患者就可能性和局限性进行清晰的沟通至关重要,这在一定程度上是为了避免做出短期内不切实际的承诺。总之,遗传学家、医疗中心和监管机构现在都面临着挑战,要开始以有效和符合伦理道德的方式将 CRISPR-Cas 技术转化为临床实践。
[Genetic therapy by CRISPR-Cas: paving the way to clinical practice].
CRISPR-Cas technology is a revolutionary technology to modify DNA sequences. Owing to its effectiveness and accuracy, CRISPR-Cas holds important promises for 'genetic therapy' for various hereditary disorders. CRISPR-Cas enables researchers to modify specific parts of the genome with unprecedented precision, which, in specific cases, can be applied to correct disease-causing DNA variants. However, several important barriers are complicating the clinical implementation trajectory. There are for example concerns regarding the safety, effectiveness and ethical justification. Nevertheless, as CRISPR-Cas becomes more widely known, doctors and healthcare providers are expected to be well aware of the developments surrounding CRISPR-Cas therapy. Professional expertise and clear communication about the possibilities and limitations to patients with genetic disorders are essential, partly to avoid making promises that are unrealistic in the short term. Altogether, geneticists, medical centers and regulators are now facing the challenges to start translating CRISPR-Cas technology into clinical practice, in an effective and ethical manner.
期刊介绍:
Het NTVG staat bekend als hét wetenschappelijke algemene medische tijdschrift. De lange historie en de degelijkheid maken het tijdschrift tot een bolwerk van medische wetenschap in druk. Ook door de goede leesbaarheid draagt het tijdschrift bij aan de voortdurende dialoog over de geneeskunde.