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Editing the Genome Ex Vivo Stem Cell Therapy

  • Genome Editing (SN Waddington and HC O'Neill, Section Editors)
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Abstract

Purpose of review

This review is focused on the recent development in the field of genomic editing of stem cells, in particular, the use of CRISPR/Cas9 systems to edit hemopoietic stem cells for beta-globinopathies and immunodeficiency syndromes. Additionally, the use of optimized genome-editing technologies in induced pluripotent stem cells will allow all affected tissue systems to be addressed.

Recent findings

The convergence of stem cell technologies and efficient genome-editing tools has brought forth the possibility of correcting human genetic diseases for the alleviation of human suffering. Meanwhile, germline editing of human zygotes is rapidly gathering pace, albeit with rising ethical and social/ moral concerns. There are remaining issues with off-target effects and cellular toxicity that needs to be addressed in full before its clinical implementation.

Summary

The era of genome-edited stem cell therapy with CRISPR/cas9 is now becoming a reality with clinical trials scheduled to start in the coming year.

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Funding

JKYC is funded by Ministry of Heath’s National Medical Research Council (NMRC), Singapore (NMRC/CSA-SI/0008/2016).

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Authors and Affiliations

  1. Department of Reproductive Medicine, KK Women’s and Children’s Hospital, 100 Bukit Timah Road, Singapore, 229899, Singapore

    Yiping Fan & Jerry Kok Yen Chan

  2. Experimental Fetal Medicine Group, Department of Obstetrics and Gynaecology, Yong Loo Lin School of Medicine, National University Health System, Singapore, 119228, Singapore

    Yiping Fan & Jerry Kok Yen Chan

  3. Obstetrics and Gynaecology, Academic Clinical Program and Stem Cell Biology Program, Duke-NUS Medical School, Singapore, 169857, Singapore

    Jerry Kok Yen Chan

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  1. Yiping Fan
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Correspondence to Yiping Fan.

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Yiping Fan and Jerry Kok Yen Chan declare that they have no conflict of interest.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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This article is part of the Topical Collection on Genome Editing

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Fan, Y., Chan, J.K.Y. Editing the Genome Ex Vivo Stem Cell Therapy. Curr Stem Cell Rep 4, 338–345 (2018). https://doi.org/10.1007/s40778-018-0148-2

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