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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الحاوية / القاعدة:	Biochemistry and Biophysics Reports
المؤلفون الرئيسيون:	Rio Miyadai, Shiori Hinata, Yuya Amemiya, Satori Shigematsu, Kazuhiro Umeyama, Hiroshi Nagashima, Kenji Yamatoya, Jun Ohgane
التنسيق:	مقال
اللغة:	الإنجليزية
منشور في:	Elsevier 2025-06-01
الموضوعات:	Epigenome editing CpG island shore FBN1 Haploinsufficiency DNA methylation dCas9-TET1
الوصول للمادة أونلاين:	http://www.sciencedirect.com/science/article/pii/S2405580825000603