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Updated: May 11, 2026

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy
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改善人类SMN2基因的TaC9-ABE介导校正

Xiaohua Peng1, Yue Chi1, Jinling Wang1

  • 1Guangdong Provincial Key Laboratory of Large Animal Models for Biomedicine, South China Institute of Large Animal Models for Biomedicine, Wuyi University, Jiangmen, China.

Biotechnology and bioengineering
|June 26, 2024
PubMed
概括

这项研究优化了脊柱肌肉缩 (SMA) 的基因编辑系统,在患者衍生细胞中实现了高效的SMN2基因校正. 改进的TaC9-ABE系统显示出作为SMA安全和有效的治疗策略的希望.

关键词:
卡斯9尼卡斯 (Cas9 nickase) 是一个腺基编辑器 腺基编辑器诱导多能干细胞的诱导干细胞.脊柱肌肉缩 脊柱肌肉缩 脊柱肌肉缩幸存者 运动神经元基因 基因转录激活器类效应器效应器.

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RNA Editing02:23

RNA Editing

RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...