Assessment and streamlined preparation of low-cytotoxicity lentiviral vectors for mobilized human hematopoietic stem cell transduction

Recombinant lentiviruses serve as important tools for diverse applications in vitro and in vivo. In research contexts, examples include ectopic protein expression [1], shRNA inhibition of targeted transcripts [2], gRNA/Cas9 CRISPR lentivirus libraries [3], and progenitor cell barcoding for lineage and tumor cell tracking studies [4,5]. In clinical trials, significant progress is being made with lentiviruses as gene therapy vectors for select inherited diseases (e.g., thalassemia, X-linked severe combined immunodeficiency [SCIDX-1]) [6,7] and for chimeric antigen receptor (CAR) T-cell engineering [8].
Source: Experimental Hematology - Category: Hematology Authors: Tags: Methods/techniques Source Type: research