[CRISPR-Cas 基因疗法:为临床实践铺平道路]。

Q4 Medicine
Philip R Jansen, Mieke van Haelst, Rob M F Wolthuis
{"title":"[CRISPR-Cas 基因疗法:为临床实践铺平道路]。","authors":"Philip R Jansen, Mieke van Haelst, Rob M F Wolthuis","doi":"","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":18903,"journal":{"name":"Nederlands tijdschrift voor geneeskunde","volume":"168 ","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"[Genetic therapy by CRISPR-Cas: paving the way to clinical practice].\",\"authors\":\"Philip R Jansen, Mieke van Haelst, Rob M F Wolthuis\",\"doi\":\"\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":18903,\"journal\":{\"name\":\"Nederlands tijdschrift voor geneeskunde\",\"volume\":\"168 \",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-08-22\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Nederlands tijdschrift voor geneeskunde\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"Medicine\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Nederlands tijdschrift voor geneeskunde","FirstCategoryId":"1085","ListUrlMain":"","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"Medicine","Score":null,"Total":0}
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。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[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.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
Nederlands tijdschrift voor geneeskunde
Nederlands tijdschrift voor geneeskunde Medicine-Medicine (all)
CiteScore
0.30
自引率
0.00%
发文量
302
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.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信