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Related Concept Videos

EPS and iPS Cells in Disease Research01:21

EPS and iPS Cells in Disease Research

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Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
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Related Experiment Video

Updated: Jun 27, 2025

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

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Innovative methodologies for rare diseases clinical trials

Rima Nabbout1,2,3, Ralf-Dieter Hilgers4

  • 1Reference centre for rare epilepsies Université Paris cité, Paris, France. rima.nabbout@aphp.fr.

Orphanet Journal of Rare Diseases
|May 7, 2024
PubMed
Summary

No abstract available in PubMed .

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