在crispr介导的基因组编辑时代治疗遗传性血液病。

IF 12.1 1区医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Molecular Therapy Pub Date : 2025-01-17 DOI:10.1016/j.ymthe.2025.01.031

Alhomidi Almotiri,Ahmed Abogosh,Ali Abdelfattah,Dalya Alowaisy,Neil P Rodrigues

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引用次数: 0

摘要

在单基因疾病的情况下，基因组编辑技术的进步原则上可以作为一种治疗策略来精确纠正受影响细胞类型中的特定基因突变并恢复功能。以β-血红蛋白病和血友病为例，我们回顾了利用crispr衍生的基因组编辑技术的最新实验突破，这些技术已经转化为遗传性血液病管理的显着改善。然而，在这些患者中使用CRISPR介导的基因组编辑也面临着挑战，我们讨论了避免这些问题以促进临床益处的可能方法。

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Treating genetic blood disorders in the era of CRISPR-mediated genome editing.

In the setting of monogenic disease, advances made in genome editing technologies can, in principle, be deployed as a therapeutic strategy to precisely correct a specific gene mutation in an affected cell type and restore functionality. Using the β-hemoglobinopathies and hemophilia as exemplars, we review recent experimental breakthroughs utilizing CRISPR-derived genome editing technology that have translated to significant improvements in the management of inherited hematologic disorders. Yet there are also challenges facing the use of CRISPR mediated genome editing in these patients and we discuss possible ways to obviate those issues for furtherance of clinical benefit.

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来源期刊

Molecular Therapy 医学-生物工程与应用微生物

CiteScore

19.20

自引率

3.20%

发文量

357

审稿时长

3 months

期刊介绍： Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.