mRNA-Driven generation of transgene-free neural stem cells from human urine-derived cells

Phil Jun Kang, Daryeon Son, Tae Hee Ko, Wonjun Hong, Wonjin Yun, Jihoon Jang, Jong Il Choi, Gwonhwa Song, Jangbo Lee, In Yong Kim, Seungkwon You

Research output: Contribution to journalArticlepeer-review

8 Citations (Scopus)


Human neural stem cells (NSCs) hold enormous promise for neurological disorders, typically requiring their expandable and differentiable properties for regeneration of damaged neural tissues. Despite the therapeutic potential of induced NSCs (iNSCs), a major challenge for clinical feasibility is the presence of integrated transgenes in the host genome, contributing to the risk for undesired genotoxicity and tumorigenesis. Here, we describe the advanced transgene-free generation of iNSCs from human urine-derived cells (HUCs) by combining a cocktail of defined small molecules with self-replicable mRNA delivery. The established iNSCs were completely transgene-free in their cytosol and genome and further resembled human embryonic stem cell-derived NSCs in the morphology, biological characteristics, global gene expression, and potential to differentiate into functional neurons, astrocytes, and oligodendrocytes. Moreover, iNSC colonies were observed within eight days under optimized conditions, and no teratomas formed in vivo, implying the absence of pluripotent cells. This study proposes an approach to generate transplantable iNSCs that can be broadly applied for neurological disorders in a safe, effcient, and patient-specific manner.

Original languageEnglish
Article number1043
Issue number9
Publication statusPublished - 2019 Sept

Bibliographical note

Publisher Copyright:
© 2019 by the authors. Licensee MDPI, Basel, Switzerland.


  • Direct conversion
  • Induced neural stemcells (iNSCs)
  • Neurological diseases
  • Reprogramming
  • Self-replicativemRNA
  • Small molecules

ASJC Scopus subject areas

  • Medicine(all)


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