Epigenome editing-mediated restoration of FBN1 expression by demethylation of CpG island shore in porcine fibroblasts

Fibrillin-1, an extracellular matrix protein encoded by the FBN1 gene, is crucial for maintaining connective tissue integrity. Mutations in FBN1 result in haploinsufficiency, leading to Marfan syndrome, in which the expression of functional FBN1 is correlated with disease onset and severity. Recent...

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Bibliographic Details

Published in:	Biochemistry and Biophysics Reports
Main Authors:	Rio Miyadai, Shiori Hinata, Yuya Amemiya, Satori Shigematsu, Kazuhiro Umeyama, Hiroshi Nagashima, Kenji Yamatoya, Jun Ohgane
Format:	Article
Language:	English
Published:	Elsevier 2025-06-01
Subjects:	Epigenome editing CpG island shore FBN1 Haploinsufficiency DNA methylation dCas9-TET1
Online Access:	http://www.sciencedirect.com/science/article/pii/S2405580825000603