Rapid and scalable personalized ASO screening in patient-derived organoids
收藏NIAID Data Ecosystem2026-05-02 收录
官方服务：
资源简介：
Targeted sequencing analysis to quantify ASO-mediated exon skipping in cardiac organoids derived from a DMD patient.
应用场景：
创建时间：
2024-10-26
相关数据集
Transcriptome sequencing of human Duchenne muscular dystrophy and CRISPR-corrected myogenic cultures during secondary myogenic differentiation.. Transcriptome sequencing of human Duchenne muscular dystrophy and CRISPR-corrected myogenic cultures during secondary myogenic differentiation.
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 Ecosystem70
Exon Exchange Approach to Repair Duchenne Dystrophin Transcripts
BackgroundTrans-splicing strategies for mRNA repair involve engineered transcripts designed to anneal target mRNAs in order to interfere with their natural splicing, giving rise to mRNA chimeras where
NIAID Data Ecosystem40
Inherited epigenetic repression of the Celf2a splicing factor mediates the amelioration of a Duchenne phenotype. Inherited epigenetic repression of the Celf2a splicing factor mediates the amelioration of a Duchenne phenotype
Exon skipping is an effective strategy for the treatment of Duchenne Muscular Dystrophy (DMD). Natural exon skipping identified in several DMD cases can help with identifying novel therapeutic tools.
NIAID Data Ecosystem10
A dystrophic Duchenne mouse model for testing human antisense oligonucleotides
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
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
