A NOTCH3 pathogenic variant influences osteogenesis and can be targeted by antisense oligonucleotides in induced pluripotent stem cells.

Lateral Meningocele Syndrome (LMS), a disorder associated with NOTCH3 pathogenic variants, presents with neurological, craniofacial and skeletal abnormalities. Mouse models of the disease exhibit osteopenia that is ameliorated by the administration of Notch3 antisense oligonucleotides (ASO) targetin...

Ausführliche Beschreibung

Bibliografische Detailangaben

Hauptverfasser:	Ernesto Canalis, Jungeun Yu, Lauren Schilling, Paymaan Jafar-Nejad, Michele Carrer
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	Public Library of Science (PLoS) 2025-01-01
Schriftenreihe:	PLoS ONE
Online-Zugang:	https://doi.org/10.1371/journal.pone.0316644
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