Gene editing is a novel gene engineeering tool that precisely modify target gene sequence. The mainly approaches of gene editing in basic and translational medicines include CRISPR/Cas9, single base editing (ABE/CBE), prime editing.The gene editing is succesfully employed in clinical trials of treatment of β-thalessemia and Duchenne muscular dystrophy.
Gene Editing Medicine
Diagram depicting CRIPR/Cas9 gene editing |
Stem cell microinjection of gene editing |
Our company aims to develope gene editing medicines for hyperlipemia, as well as various genetic diseases.
PCSK9 is a hot target for hypercholesterolemia. The current medicine in markets mostly are antibody or SiRNA for PCSK9.
Our company is developing RNA editing tools for PCSK9 to treat hypercholesterolemia.