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Antisense Oligonucleotide Rescue of CGG Expansion-Dependent FMR1 Mis-Splicing in Fragile X Syndrome Restores FMRP

NIAID Data Ecosystem2026-05-01 收录

脆性X综合征基因治疗

FMR1剪接调控

数据链接：

https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE202179 数据链接链接失效反馈

官方服务：

资源简介：

This SuperSeries is composed of the SubSeries listed below. Refer to individual Series

应用场景：

创建时间：

2023-09-14

脆性X综合征基因治疗

AAV载体递送DGKk

Fragile X syndrome (FXS) is the most frequent form of familial intellectual disability. FXS results from the lack of the RNA binding protein FMRP and is associated with the deregulation of signaling p

NIAID Data Ecosystem80

Targeted reactivation of FMR1 transcription in FXS embryonic stem cells

脆性X综合征基因治疗

CRISPR转录激活

The aim of this work is to selectively increase transcription from the FMR1 locus in FXS hESC lines using CRISPR-Cas9 fused to transcriptional activators. Endonuclease deficient Cas9 (dCas9) was fused

NIAID Data Ecosystem50

Table_1_Targeted Reactivation of FMR1 Transcription in Fragile X Syndrome Embryonic Stem Cells.XLSX

脆性X综合征基因治疗

CRISPR基因激活

Fragile X Syndrome (FXS) is the most common inherited cause of intellectual disability and autism. It results from expansion of a CGG nucleotide repeat in the 5′ untranslated region (UTR) of FMR1. Lar

NIAID Data Ecosystem30

Rescue of Fragile X syndrome neurons by DNA methylation editing of the FMR1 gene [methylation]. Rescue of Fragile X syndrome neurons by DNA methylation editing of the FMR1 gene [methylation]

脆性X综合征基因治疗

DNA甲基化

Fragile X syndrome (FXS), the most common genetic form of intellectual disability in male, is caused by silencing of the FMR1 gene by hypermethylation of the CGG expansion mutation in the 5’UTR region

NIAID Data Ecosystem60

Rescue of Fragile X syndrome neurons by DNA methylation editing of the FMR1 gene [methylation]

脆性X综合征基因治疗

DNA甲基化

NIAID Data Ecosystem40