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Updated: Jul 13, 2026

Primordial Germ Cell Transplantation for CRISPR/Cas9-based Leapfrogging in Xenopus
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Published on: February 1, 2018

通过核移植和结合细胞和基因疗法纠正遗传缺陷

William M Rideout1, Konrad Hochedlinger, Michael Kyba

  • 1Whitehead Institute for Biomedical Research, 9 Cambridge Center, Cambridge, MA 02142, USA.

Cell
|April 17, 2002
PubMed
概括

这项研究展示了一种新的基因治疗方法,通过修复胚胎干细胞中的Rag2基因来治疗免疫缺陷. 这种方法成功地恢复了小鼠的免疫功能,为遗传疾病提供了潜在的治疗方法.

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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine09:54

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