High-throughput sequencing data of eeCas9 and eeBE of file7
收藏NIAID Data Ecosystem2026-03-14 收录
下载链接：
https://www.ncbi.nlm.nih.gov/sra/SRP340619
下载链接
链接失效反馈官方服务：
资源简介：
Delivering AAV8-eeCas9 into mice liver to target PCSK9 gene for curing disease.
应用场景：
创建时间：
2022-10-01
相关数据集
Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells
Fanconi Anemia (FA) is a debilitating genetic disorder with a wide range of severe symptoms including bone marrow failure and predisposition to cancer. CRISPR-Cas genome editing manipulates genotypes
NIAID Data Ecosystem70
In vivo adenine base editing of PCSK9 in mice and macaques reduces LDL-cholesterol levels [amplicon-seq]
The majority of known pathogenic point mutations in the human genome are Câ¢G to Tâ¢A substitutions. Adenine base editors (ABEs), comprised of nuclease-impaired Cas9 fused to adenine deaminases, ena
NIAID Data Ecosystem60
In vivo genome editing restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers [RNA-seq]. In vivo genome editing restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers [RNA-seq]
Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease. Clustered regularly interspaced short palindromic repeat (CRISPR)-mediated gene editing is a promising strategy for permanen
NIAID Data Ecosystem50
一种基于针对特定基因反向互补序列和Py位点调控可变剪切的方法
本发明公布了一种基于针对特定基因反向互补序列和Py位点调控可变剪切的方法。具体是针对特定靶标基因可变剪切外显子上下游内内含子设计反向互补序列，并和Py位点一起构建进质粒中，通过反向互补序列与靶标基因配对，使其靶向定位，并通过Py区招募U2AF65蛋白和其他剪切因子，从而对靶标基因的可变剪切产生调节作用。本发明的方法可用于制备药物，在治疗由于可变剪切错误引起的人类疾病中有着极为重要的意义。
中国科学院中国科学技术大学科学数据中心60
The specificity of CCRS regulating target genes transcription-1
In this study, we fused CasRx crRNA with antisense ribozyme to construct a synthetic fusion guide RNA that can interact with both CasRx protein and ribozyme, and tested the ability of this approach in
NIAID Data Ecosystem50
