官方服务:
资源简介:
Reactivation of ?-globin Expression through Cas9 or Base Editor to Treat Ã-Hemoglobinopathies
应用场景:
创建时间:
2019-08-03
相关数据集
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
This SuperSeries is composed of the SubSeries listed below. Overall design: Refer to individual Series
NIAID Data Ecosystem100
Precise correction of heterozygous SHOX2 mutations in hiPSCs derived from patients with atrial fibrillation via genome editing and sib-selection
Patient-specific human induced pluripotent stem cells (hiPSCs) offer unprecedented opportunities for the investigation of multigenic disease, personalized medicine, and stem cell therapy. For heteroge
NIAID Data Ecosystem50
Treatment of a metabolic liver disease by in vivo prime editing in mice
Prime editing is a highly versatile CRISPR-based genome editing technology with the potential to correct the vast majority of genetic defects1. However, correction of a disease phenotype in vivo in so
NIAID Data Ecosystem40
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
Measurement of base substitution induced by adenine base editor
NIAID Data Ecosystem50
Ex vivo therapeutic base and prime editing using chemically derived hepatic progenitors in a mouse model of tyrosinemia type 1. Kim et al.
Bipotent differentiation capacity of HT1-mCdHs. (A) Gene expression levels of mature hepatocyte-specific markers determined by RT-qPCR. Gapdh was used as an internal control. Data are mean ± SD (n=9).
NIAID Data Ecosystem50



