High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing.

ABSTRACT: Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) has shown clear neurological benefit in rare diseases, which is achieved through the engraftment of genetically modified microglia-like cells (MLCs) in the brain. Still, the engraftment dynamics and the nature of engineered MLCs, as well as their potential use in common neurogenerative diseases, have remained largely unexplored. Here, we comprehensively characterized how different routes of administration affect the biodistribution of genetically engineered MLCs and other HSPC derivatives in mice. We generated a high-resolution single-cell transcriptional map of MLCs and discovered that they could clearly be distinguished from macrophages as well as from resident microglia by the expression of a specific gene signature that is refle

SUBMITTER: Plasschaert RN 

PROVIDER: S-EPMC9552809 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature