Patient-derived isogenic iPSCs
نویسندگان
چکیده
منابع مشابه
Molecular and functional resemblance of differentiated cells derived from isogenic human iPSCs and SCNT-derived ESCs.
Patient-specific pluripotent stem cells (PSCs) can be generated via nuclear reprogramming by transcription factors (i.e., induced pluripotent stem cells, iPSCs) or by somatic cell nuclear transfer (SCNT). However, abnormalities and preclinical application of differentiated cells generated by different reprogramming mechanisms have yet to be evaluated. Here we investigated the molecular and func...
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Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by mutations of the survival of motor neuron 1 (SMN1) gene. In the pathogenesis of SMA, pathological changes of the neuromuscular junction (NMJ) precede the motor neuronal loss. Therefore, it is critical to evaluate the NMJ formed by SMA patients' motor neurons (MNs), and to identify drugs that can restore the normal condition. We...
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ژورنال
عنوان ژورنال: Nature Genetics
سال: 2011
ISSN: 1061-4036,1546-1718
DOI: 10.1038/ng.931