Viruses and virally-derived particles have the intrinsic capacity to deliver molecules to cells, but the difficulty of readily altering cell-type selectivity has hindered their use for therapeutic del

NIAID Data Ecosystem40

In vivo adenine base editing of PCSK9 in mice and macaques reduces LDL-cholesterol levels [RNA-seq]

体内基因编辑

PCSK9靶向治疗

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 Ecosystem50

Study on genome editing tool

体内基因编辑

基因工程工具开发

To develop a genome tool with high efficiency in in vivo study

NIAID Data Ecosystem40