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- W2567664129 abstract "Duchenne muscular dystrophy (DMD) affects approximately 1 in 5000 children and is characterized by progressive muscle wasting, weakness and premature death. DMD is caused by mutations in the gene encoding dystrophin, which plays an essential role in maintaining muscle integrity by providing a structural link between the cytoskeleton and the extracellular matrix. In the absence of dystrophin, affected muscles experience repeated bouts of injury, inflammation and necrosis, resulting in reduced regenerative capacity and the replacement of muscle mass with fibrotic and adipose tissue. Both gene and cell therapies are candidate approaches to replace the defective dystrophin gene. While gene therapy has the potential to halt disease progression in targeted muscles, cell therapies could both introduce replacement genes and generate new muscle or muscle stem cells to affected tissues. Cell therapies for DMD have thus far shown moderate success due to limited ex vivo expansion potential of progenitor cells, poor survival and migration of cells following transplantation, inefficient myogenic conversion of non-myogenic cells as well as host rejection of donor cells. Most of these limitations could potentially be addressed using gene corrected induced pluripotent stem (iPS) cells derived from biopsies of affected patients.We have established and characterized several iPSC lines from control, dystrophic (mdx) and transgenic-mdx mice. Selected iPS cells expressed markers associated with pluripotency, exhibited normal karyotypes and were able to form all three germ layers during in vivo teratoma assays. To generate myogenic progenitors from iPS cells, we have investigated the use of an inducible form of the myogenic regulatory factor MyoD (MyoD-ER[T]). Upon transient activation of MyoD-ER[T], iPSCs are readily converted into myogenic progenitor cells that can efficiently differentiate into myocytes and fuse into myotubes in vitro. iPS cells derived from dystrophic mice and which carry different versions of dystrophin expression cassettes can engraft into mdx muscles in vivo and generate dystrophin-positive myofibers. We are currently comparing the efficiency of various myogenic cell populations resulting from MyoD-ER[T] induction to generate functional myogenic engraftment in vivo following transplantation into mdx mouse hosts. We are also comparing the relative efficiency of dystrophin production in myogenic cells derived from mdx iPSCs using gene replacement vs. gene modification approaches. Together, these approaches show potential for ex vivo gene therapy of DMD using dystrophin-corrected autologous cell transplantation." @default.
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- W2567664129 date "2015-05-01" @default.
- W2567664129 modified "2023-09-24" @default.
- W2567664129 title "613. Characterization of IPSC-Derived Myogenic Progenitors Isolated from Mouse Models of Duchenne Muscular Dystrophy" @default.
- W2567664129 doi "https://doi.org/10.1016/s1525-0016(16)34222-8" @default.
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