Development of iPSC-based clinical trial selection platform for patients with ultrarare diseases
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DOI: 10.1126/sciadv.abl4370
Abstract: A “Leap-of-Faith” approach is used to treat patients with previously unknown ultrarare pathogenic mutations, often based on evidence from patients having dissimilar but more prevalent mutations. This uncertainty reflects the need to develop personalized prescreening… read more here.
Keywords: platform; patient; development ipsc; clinical trial ... See more keywords