Genomic DNA of autologous CD34+ hematopoietic stem and progenitor cells (CD34+ HSPC genomic DNA)

The genomic DNA of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) serves as the primary substrate for ex vivo gene therapies and genome editing (StatPearls, 2023). These cells are multipotent progenitors capable of self-renewal and differentiating into all mature blood cell types, including erythrocytes, leukocytes, and platelets (NIH, 2024). In patients with genetic blood disorders or metabolic diseases, the genomic DNA of these cells contains pathogenic mutations that impair normal physiological function. Therapeutic intervention involves harvesting these cells from the patient, followed by the introduction of functional gene copies via lentiviral vectors or the precise modification of specific loci using CRISPR-Cas9 technology (FDA, 2023). Once modified, the cells are re-infused into the patient, where they engraft in the bone marrow and produce a progeny of healthy, functional blood cells. This approach provides a potentially curative treatment by addressing the underlying genetic cause of diseases such as sickle cell disease and beta-thalassemia (Nature Medicine, 2022; PubMed, 2021).

Other names

Autologous CD34+ cellsHematopoietic stem and progenitor cellsHSPCsCD34+ cellsHematopoietic stem cell genome

Ex vivo genetic modification of hematopoietic stem cells via viral vector-mediated gene addition or CRISPR-Cas9-mediated gene editing to restore protein function or alter gene expression (FDA, 2023; Nature Medicine, 2022).