CD117-targeted LNP-Cas9

**CD117-targeted LNP-Cas9** is an experimental, antibody-targeted lipid nanoparticle platform for in vivo delivery of mRNA-encoded genome-editing cargoes to hematopoietic stem and progenitor cells. Anti-CD117 antibodies on the nanoparticle surface bind the stem cell factor receptor c-Kit on hematopoietic stem cells, enabling delivery of an adenine base editor mRNA and an HBB sickle-mutation-targeting single-guide RNA. In preclinical studies, the editing formulation converted the pathogenic sickle-cell HBB allele toward the nonpathogenic HBB Makassar variant in patient-derived hematopoietic cells; related CD117/LNP formulations delivered pro-apoptotic PUMA mRNA to provide non-genotoxic conditioning before hematopoietic stem cell transplantation. It is a research-stage platform rather than a clinically named drug product or an INN-assigned medicine. ([pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/37499029/))

Other names

anti-human CD117/LNP-based editing systemCD117/LNPCD117/LNP-mRNACD117-targeted LNP-Cas9CD-117-targeted LNP-Cas9CD 117-targeted LNP-Cas9