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.
journal_name
Med Hypothesesjournal_title
Medical hypothesesauthors
Hagan M,Ashraf M,Kim IM,Weintraub NL,Tang Ydoi
10.1016/j.mehy.2017.11.009subject
Has Abstractpub_date
2018-01-01 00:00:00pages
97-100eissn
0306-9877issn
1532-2777pii
S0306-9877(17)31104-0journal_volume
110pub_type
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