Generation of corrected hiPSC clones from a Cornelia de Lange Syndrome (CdLS) patient through CRISPR-Cas-based technology

Generation of corrected hiPSC clones from a Cornelia de Lange Syndrome (CdLS) patient through CRISPR-Cas-based technology

Abstract Background Cornelia de Lange syndrome (CdLS) is a rare multisystem genetic disorder which is caused by genetic defects involving the Nipped-B-like protein (NIPBL) gene in the majority of clinical cases (60–70%). Currently, there are no specific cures available for CdLS and clinical manageme...

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Main Authors: Alessandro Umbach, Giulia Maule, Eyemen Kheir, Alessandro Cutarelli, Marika Foglia, Luca Guarrera, Luca L. Fava, Luciano Conti, Enrico Garattini, Mineko Terao, Anna Cereseto
Format: Article
Language:English
Published: BMC 2022-09-01
Series:Stem Cell Research & Therapy
Subjects:
Cornelia de Lange Syndrome (CdLS)
CRISPR-Cas9
Homology Directed Repair (HDR)
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Prime editor
Patient-derived hiPSCs
Online Access:https://doi.org/10.1186/s13287-022-03135-0

Internet

https://doi.org/10.1186/s13287-022-03135-0

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