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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

NIAID Data Ecosystem2026-05-02 收录

基因编辑治疗

镰状细胞病治疗

数据链接：

https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE275158 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：

2024-09-23

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

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

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

Sickle Cell Disease Treatment Market Size Worth $6.94 Billion By 2030 | CAGR: 18.8%

镰状细胞病治疗

市场分析

The global sickle cell disease treatment market size is expected to reach USD 6.94 billion by 2030, according to a new study by Polaris Market Research.

www.polarismarketresearch.com2023-10-19 更新60

Base editor mediated correction of a tau mutation rescues cognitive decline in a mouse model of Alzheimers disease. Homo Sapiens

基因编辑治疗

阿尔茨海默病模型

Low sequence reads

NIAID Data Ecosystem40