Senescence and inflammation are unintended adverse consequences of CRISPR-Cas9/AAV6 mediated gene editing in hematopoietic stem cells

NIAID Data Ecosystem2026-05-02 收录

基因编辑治疗

造血干细胞工程

数据链接：

https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE244257 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

创建时间：

2025-06-05

Precise genomic editing of a pathogenic RBM20 mutation rescues dilated cardiomyopathy. Precise genomic editing of a pathogenic RBM20 mutation rescues dilated cardiomyopathy

基因编辑治疗

扩张型心肌病

Mutations in RNA binding motif protein 20 (RBM20) are a common cause of dilated cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine rich (RS-rich) domain, resulting in mis-loc

NIAID Data Ecosystem120

Efficient in vivo Genome Editing Prevents Hypertrophic Cardiomyopathy in Mice [Amplified_cDNA]. Efficient in vivo Genome Editing Prevents Hypertrophic Cardiomyopathy in Mice [Amplified_cDNA]

基因编辑治疗

肥厚型心肌病动物模型

Dominant pathogenic variants encoding amino acid substitutions in cardiac myosin heavy chain cause hypertrophic cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, hea

NIAID Data Ecosystem50

In vivo Ryr2 editing corrects catecholaminergic polymorphic ventricular tachycardia

基因编辑治疗

遗传性心律失常

Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a lethal inherited arrhythmia disorder most commonly caused by missense mutations in the RyR2 gene. The goal of this study was to determ

NIAID Data Ecosystem20

Gene Editing of Hematopoietic Stem Cells Restores T Cell Response in a Familial Hemophagocytic Lymphohistiocytosis Model. Gene Editing of Hematopoietic Stem Cells Restores T Cell Response in a Familial Hemophagocytic Lymphohistiocytosis Model

基因编辑治疗

免疫缺陷疾病

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

NIAID Data Ecosystem100

Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells. Homo sapiens

基因编辑治疗

囊性纤维化基因校正

A systematic implementation of six efficiency optimizations for prime editing led to high levels of functional correction, in airway epithelial cells, of the predominant mutation that causes cystic fi

NIAID Data Ecosystem30

Senescence and inflammation are unintended adverse consequences of CRISPR-Cas9/AAV6 mediated gene editing in hematopoietic stem cells

资源简介：

相关数据集