CD90-targeted lentiviral vectors for HSC gene therapy.

ABSTRACT: Hematopoietic stem cell (HSC) gene therapy is currently performed on CD34⁺ hematopoietic stem and progenitor cells containing less than 1% true HSCs and requiring a highly specialized infrastructure for cell manufacturing and transplantation. We have previously identified the CD34⁺CD90⁺ subset to be exclusively responsible for short- and long-term engraftment. However, purification and enrichment of this subset is laborious and expensive. HSC-specific delivery agents for the direct modification of rare HSCs are currently lacking. Here, we developed novel targeted viral vectors to specifically transduce CD90-expressing HSCs. Anti-CD90 single chain variable fragments (scFvs) were engineered onto measles- and VSV-G-pseudotyped lentiviral vectors that were knoc

SUBMITTER: Berckmueller K 

PROVIDER: S-EPMC10556220 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature