CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题：CRISPR-based precision medicine for hematologic disorders: Advancements, challenges, and prospects.
CRISPR-based precision medicine for hematologic disorders: Advancements, challenges, and prospects.
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能够在基因组序列中引入明确改变的可编程核酸酶，是精准医疗的重要工具。尽管锌指核酸酶（ZFN）、转录激活因子样效应物核酸酶（TALEN）和巨型核酸酶等技术均已得到研究，CRISPR/Cas9技术的出现彻底改变了基因组工程领域。CRISPR/Cas9除用于疾病建模外，还推动了更安全有效的血液系统疾病治疗策略和个体化T细胞疗法发展。本文讨论CRISPR技术在血液系统疾病治疗中的应用、疗效及正在进行的临床试验，分析其成功应用面临的障碍和已探索的克服方法，并提出进一步改进该基因编辑工具以实现靶向治疗的思路。
The development of programmable nucleases to introduce defined alterations in genomic sequences has been a powerful tool for precision medicine. While several nucleases such as zinc-finger nucleases (ZFN), transcriptor activator-like effector nucleases (TALEN), and meganucleases have been explored, the advent of CRISPR/Cas9 technology has revolutionized the field of genome engineering.
In addition to disease modeling, the CRISPR/Cas9 technology has contributed to safer and more effective treatment strategies for hematologic diseases and personalized T-cell-based therapies.
Here we discuss the applications of the CRISPR technology in the treatment of hematologic diseases, their efficacy, and ongoing clinical trials.
We examine the obstacles to their successful use and the approaches investigated to overcome these challenges.
Finally, we provide our perspectives to improve this genome editing tool for targeted therapies.
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