Purification of Human CD34⁺CD90⁺ HSCs Reduces Target Cell Population and Improves Lentiviral Transduction for Gene Therapy.

ABSTRACT: Hematopoietic stem cell (HSC) gene therapy has the potential to cure many genetic, malignant, and infectious diseases. We have shown in a nonhuman primate gene therapy and transplantation model that the CD34⁺CD90⁺ cell fraction was exclusively responsible for multilineage engraftment and hematopoietic reconstitution. In this study, we show the translational potential of this HSC-enriched CD34 subset for lentivirus-mediated gene therapy. Alternative HSC enrichment strategies include the purification of CD133⁺ cells or CD38^low/- subsets of CD34⁺ cells from human blood products. We directly compared these strategies to the isolation of CD90⁺ cells using a good manufacturing practice (GMP) grade flow-sorting protocol with clinic

SUBMITTER: Radtke S 

PROVIDER: S-EPMC7424231 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature