官方服务：
资源简介：
To develop a genome tool with high efficiency in in vivo study
应用场景：
创建时间：
2023-02-03
相关数据集
In vivo base editing extends lifespan of a humanized mouse model of prion disease
Prion disease is a fatal neurodegenerative disease caused by the misfolding of prion protein (PrP) encoded by the PRNP gene. While there is currently no cure for the disease, depleting PrP in the brai
NIAID Data Ecosystem90
Small Cas13 proteins enable compact RNA base editors
CRISPR-Cas13 systems have been developed for precise RNA editing, an attractive therapeutic strategy when temporary changes are desirable or DNA editing is not possible. We identified and characterize
NIAID Data Ecosystem40
Outcomes of intrahepatic cholangiocarcinoma specific CRISPR screening in vivo [RNA-seq]
We report the genertion of a gRNA library that induces mutations often found in intrahepatic cholangiocarcinoma. We use this to screen in vivo mutations that when lost act synergistically with mutant
NIAID Data Ecosystem30
PpCas9 from Pasteurella pneumotropica - a compact Type II-C Cas9 ortholog active in human cells. Part 2
Characterization Type II-C CRISPR Cas systems from Defluviimonas sp. 20V17 and Pasteurella pneumotropica. Illumina sequencing reads from PpCas9 specificity tests.
NIAID Data Ecosystem70
In vivo base editing by a single intravenous vector injection for the treatment of hemoglobinopathies
Individuals with beta-thalassemia or Sickle Cell Disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% HbF appear to be symptom-free. Here, we used a non-integrating HDAd5/35++
NIAID Data Ecosystem30
