Effective regeneration of dystrophic muscle using autologous iPSC-derived progenitors with CRISPR-Cas9 mediated precise correction

Mackenzie Hagan, Muhammad Ashraf, Il man Kim, Neal L. Weintraub, Yaoliang Tang

Research output: Contribution to journalArticle

6 Scopus citations

Abstract

Duchenne muscular dystrophy (DMD) is a lethal muscle wasting disease caused by a lack of dystrophin, which eventually leads to apoptosis of muscle cells and impaired muscle contractility. Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9 (CRISPR/Cas9) gene editing of induced pluripotent stem cells (IPSC) offers the potential to correct the DMD gene defect and create healthy IPSC for autologous cell transplantation without causing immune activation. However, IPSC carry a risk of tumor formation, which can potentially be mitigated by differentiation of IPSC into myogenic progenitor cells (MPC). We hypothesize that precise genetic editing in IPSC using CRISPR-Cas9 technology, coupled with MPC differentiation and autologous transplantation, can lead to safe and effective muscle repair. With future research, our hypothesis may provide an optimal autologous stem cell-based approach to treat the dystrophic pathology and improve the quality of life for patients with DMD.

Original languageEnglish (US)
Pages (from-to)97-100
Number of pages4
JournalMedical Hypotheses
Volume110
DOIs
StatePublished - Jan 2018

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Keywords

  • CRISPR-Cas9
  • DMD
  • Myogenesis
  • Myogenic progenitor cells
  • Precise correction

ASJC Scopus subject areas

  • Medicine(all)

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