Evaluation of single cut sgRNA for dystrohpin restoration
收藏NIAID Data Ecosystem2026-03-11 收录
官方服务:
资源简介:
Develop a method for sensitive valuation of dystrophin restoring sgRNA
应用场景:
创建时间:
2020-02-28
相关数据集
DataSheet1_Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.pdf
Pyruvate kinase deficiency (PKD) is an autosomal recessive disorder caused by mutations in the PKLR gene. PKD-erythroid cells suffer from an energy imbalance caused by a reduction of erythroid pyruvat
NIAID Data Ecosystem210
Additional file 2 of Engineered virus-like particle-assembled Vegfa-targeting Cas9 ribonucleoprotein treatment alleviates neovascularization in wet age-related macular degeneration
Additional file 2: Table S1. Oligomers for sgRNA cloning and primers used to amplify the on-target site for NGS. Table S2. Primers used for amplification of off-target sites: Off-targets 1–4 (CCTop-pr
NIAID Data Ecosystem70
Base editor mediated correction of a tau mutation rescues cognitive decline in a mouse model of Alzheimers disease. Homo Sapiens
Low sequence reads
NIAID Data Ecosystem60
In vivo selection for corrected ß-globin alleles after CRISPR/Cas9 editing in human sickle hematopoietic stem cells. Homo sapiens
Sickle Cell Disease (SCD), one of the world's most common genetic disorders, causes anemia and progressive multiorgan damage that typically shortens lifespan by decades; currently there is no broadly
NIAID Data Ecosystem130
Universal correction of blood coagulation factor VIII in patient-derived induced pluripotent stem cells using CRISPR/Cas9
Confirmation of the nuclease acivities at the On- and Off-target sites; Hemophilia A (HA) is caused by genetic mutations in the blood coagulation factor VIII (FVIII). Genome editing approaches can be
NIAID Data Ecosystem70



