| Autor: |
Xie N; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA., Chu SN; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA., Schultz CB; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA., Chan SSK; Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA.; Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USA.; Lillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USA. |
| Abstrakt: |
Differentiation of pluripotent stem cells (PSCs) is a promising approach to obtaining large quantities of skeletal myogenic progenitors for disease modeling and cell-based therapy. However, generating skeletal myogenic cells with high regenerative potential is still challenging. We recently reported that skeletal myogenic progenitors generated from mouse PSC-derived teratomas possess robust regenerative potency. We have also found that teratomas derived from human PSCs contain a skeletal myogenic population. Here, we showed that these human PSC-derived skeletal myogenic progenitors had exceptional engraftability. A combination of cell surface markers, CD82, ERBB3, and NGFR enabled efficient purification of skeletal myogenic progenitors. These cells expressed PAX7 and were able to differentiate into MHC+ multinucleated myotubes. We further discovered that these cells are expandable in vitro. Upon transplantation, the expanded cells formed new dystrophin + fibers that reconstituted almost ¾ of the total muscle volume, and repopulated the muscle stem cell pool. Our study, therefore, demonstrates the possibility of producing large quantities of engraftable skeletal myogenic cells from human PSCs. |
