Targeted allele-specific FGFR2 knockdown via human recombinant ferritin nanoparticles for personalized treatment of Crouzon syndrome

Targeted allele-specific FGFR2 knockdown via human recombinant ferritin nanoparticles for personalized treatment of Crouzon syndrome

Crouzon syndrome is a rare genetic craniofacial malformation caused by heterozygous gain-of-function mutations in the FGFR2 gene. The resulting constitutive activation of the FGFR2 signaling causes the premature osteogenic differentiation of calvarial mesenchymal stromal cells in skull sutures, lead...

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Main Authors: Federica Tiberio, Martina Salvati, Luca Polito, Giada Tisci, Alessia Vita, Ornella Parolini, Luca Massimi, Lorena Di Pietro, Pierpaolo Ceci, Gianpiero Tamburrini, Alessandro Arcovito, Elisabetta Falvo, Wanda Lattanzi
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
craniosynostosis
Crouzon syndrome
small interfering RNA
ferritin nanoparticles
gene silencing
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124003147
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