2014
DOI: 10.1016/j.bbrc.2014.04.084
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Abstract: In this study, we induced differentiation of CPT II-deficient hiPSCs into mature myocytes in a highly efficient and reproducible manner and recapitulated some aspects of the disease phenotypes of CPT II deficiency in the myocyte disease models. This approach addresses the challenges of modeling the abnormality of FAO in CPT II deficiency using iPSC technology and has the potential to revolutionize translational research in this field.

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Cited by 46 publications
(30 citation statements)
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“…Our studies employed acylcarnitine administration externally to cells, which were in line with the high concentrations of long-chain acylcarnitines within plasma and muscle under conditions of FAOD (23,43). .…”
Section: Discussionmentioning
confidence: 87%
“…Our studies employed acylcarnitine administration externally to cells, which were in line with the high concentrations of long-chain acylcarnitines within plasma and muscle under conditions of FAOD (23,43). .…”
Section: Discussionmentioning
confidence: 87%
“…MyoD-reprogrammed hPSCs can generate myotubes within 10 days (Rao et al, 2012) and are also able to engraft when transplanted in mouse muscle (Goudenege et al, 2012;Ozasa et al, 2007;Zheng et al, 2006) (Table S1). Moreover, MyoDreprogrammed myotubes have been used to model muscle diseases such as Duchenne or Miyoshi muscular dystrophies (Shoji et al, 2015;Yasuno et al, 2014;Young et al, 2016) (Table S1). …”
Section: Direct Reprogramming Strategies Using Transcription Factorsmentioning
confidence: 99%
“…With respect to medication therapy, Bonnefont et al reported that bezafibrate increased the enzymatic activity of CPT II in patients with CPT II deficiency over a long-term follow-up (7). Moreover, Yasuno et al demonstrated an increased enzymatic activity following bezafibrate treatment in human induced pluripotent stem cell-derived myocytes obtained from a Japanese patient with CPT II deficiency (8). In contrast, Ørngreen et al found no significant improvement of bezafibrate treatment in a randomized clinical trial of patients with CPT II deficiency and very long-chain acyl-CoA dehydrogenase deficiencies, which put into question the effectiveness of bezafibrate in vitro and in vivo (9).…”
Section: Discussionmentioning
confidence: 98%