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Updated: Jun 19, 2026

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development
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开发基因编辑协议的多维响应表面方法 p67phox-缺陷慢性粒状瘤疾病的基因编辑协议

Thomas E Whittaker1, Shefta E Moula1, Sameer Bahal1

  • 1Infection, Immunity, and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.

Human gene therapy
|December 8, 2023
PubMed
概括

使用CRISPR/Cas9和腺相关病毒 (AAV) 进行基因编辑可以纠正遗传性血液疾病. 一种新的响应表面方法优化了造血干细胞中的基因校正,平衡了治疗性基因插入与细胞活性的作用.

关键词:
在 AAV AAV AAV 中.在CGD上,你会看到CGD.基因编辑 基因编辑同质性导向的维修是指导维修.响应表面方法的方法.

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