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

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 management is ne...

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Detalles Bibliográficos
Autores principales: Umbach, Alessandro, Maule, Giulia, Kheir, Eyemen, Cutarelli, Alessandro, Foglia, Marika, Guarrera, Luca, Fava, Luca L., Conti, Luciano, Garattini, Enrico, Terao, Mineko, Cereseto, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9438151/
https://www.ncbi.nlm.nih.gov/pubmed/36056433
http://dx.doi.org/10.1186/s13287-022-03135-0