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Updated: Jul 25, 2026

Highly Efficient Gene Disruption of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9
08:27

Highly Efficient Gene Disruption of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9

Published on: April 10, 2018

构建一个功能性的人类抑制器tRNA基因:一种针对β-thalassaemia的基因治疗方法

G F Temple, A M Dozy, K L Roy

    Nature
    |April 8, 1982
    PubMed
    概括

    研究人员设计了一个人类氨酸转移RNA (tRNA) 基因,以抑制珀色无意义突变. 这种修改后的tRNA成功地纠正了βO血症mRNA中的特定突变,为突变检测和基因疗法提供了潜力.

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    Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells

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