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Development of iPSC-based clinical trial selection platform for patients with ultrarare diseases
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 platforms for these patients to a...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Association for the Advancement of Science
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8993122/ https://www.ncbi.nlm.nih.gov/pubmed/35394834 http://dx.doi.org/10.1126/sciadv.abl4370 |