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A novel IRES identified in DMD results in a functional N-truncated dystrophin, providing a potential route to therapy for patients with 5’ mutations.

ChEBI2014-08-02 更新2026-07-21 收录

杜氏肌营养不良基因治疗

IRES介导的蛋白表达

数据链接：

https://www.ebi.ac.uk/biostudies/ArrayExpress/studies/E-GEOD-56148 数据链接链接失效反馈

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A novel IRES identified in DMD results in a functional N-truncated dystrophin, providing a potential route to therapy for patients with 5’ mutations.

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创建时间：

2014-08-02

杜氏肌营养不良基因治疗

成纤维细胞转录组学

We sought to investigate whether the reduced differentiation and fusion competence in DMD-R3381X cultures was due to aberrant myogenic gene expression. We performed transcriptome sequencing and analys

NIAID Data Ecosystem80

A dystrophic Duchenne mouse model for testing human antisense oligonucleotides

杜氏肌营养不良基因治疗

反义寡核苷酸临床前评估

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease generally caused by reading frame disrupting mutations in the DMD gene resulting in loss of functional dystrophin protein. The read

NIAID Data Ecosystem70

Targeting Duchenne Muscular Dystrophy by Skipping DMD Exon 45 with Base Editors

杜氏肌营养不良基因治疗

碱基编辑外显子跳过

Duchenne muscular dystrophy is an X-linked monogenic disease caused by mutations in the dystrophin gene (DMD) and characterized by progressive muscle weakness leading to loss of ambulation and signifi

NIAID Data Ecosystem40

In vivo genome editing restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers [Amplicon-seq]

杜氏肌营养不良基因治疗

CRISPRi基因编辑

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease. Clustered regularly interspaced short palindromic repeat (CRISPR)-mediated gene editing is a promising strategy for permanen

NIAID Data Ecosystem80

Gene expression profiling of Galgt2 overexpression in mdx skeletal muscle. Mus musculus

杜氏肌营养不良基因治疗

大鼠转录组学

Transgenic overexpression of Galgt2 in the skeletal muscles of mdx mice inhibits the development of disease pathology associated with muscular dystrophy. This is the case both in transgenic mice, wher

NIAID Data Ecosystem40

A novel IRES identified in DMD results in a functional N-truncated dystrophin, providing a potential route to therapy for patients with 5’ mutations.

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