Multi-level omics analysis of dystrophin loss and therapeutic restoration in a murine model

ChEBI2015-09-24 更新2026-07-12 收录

杜氏肌营养不良症

多组学治疗评估

数据链接：

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Multi-level omics analysis of dystrophin loss and therapeutic restoration in a murine model

创建时间：

2015-09-24

Data from: Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

杜氏肌营养不良症

临床试验

Objective: We carried out first-in-patient studies of vamorolone, a first-in-class dissociative steroidal anti-inflammatory drug, in Duchenne muscular dystrophy. Methods: An open-label, multiple-asc

DataCite Commons2025-06-01 更新120

Mass-spectrometry results from fibroadipogenic progenitor cells isolated from healthy controls (n=3) and DMD patients (n=3)

蛋白质组学

杜氏肌营养不良症

Duchenne muscular dystrophy is a genetic disease produced by mutations in the dystrophin gene characterized by early onset muscle weakness leading to severe and irreversible disability. Muscle degener

DataCite Commons2025-03-25 更新90

Multi-level omics analysis of gene expression in a murine model of dystrophin loss and therapeutic restoration [mRNA]

杜氏肌营养不良症

基因治疗疗效评估

Duchenne muscular dystrophy (DMD) is a classical monogenic disorder, a model disease for genomic studies and a priority candidate for regenerative medicine and gene therapy. Although the genetic cause

NIAID Data Ecosystem40

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

基因治疗疗效评估

杜氏肌营养不良症

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 Ecosystem110

Dataset Longitudinal alterations of gait features in children with Duchenne muscular dystrophy

步态分析

杜氏肌营养不良症

This dataset contains subject information, 3D gait analysis data (spatiotemporal, kinematic and kinetic parameters) of children with Duchenne muscular dystrophy and typically developing children. This

DataCite Commons2025-06-01 更新60

Multi-level omics analysis of dystrophin loss and therapeutic restoration in a murine model

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