Hematopoietic stem cell gene editing and expansion: state-of-the-art technologies and recent applications

Hematopoietic stem cells (HSCs) are a rare bone marrow (BM) population of cells that are multipotent and able to self-renew, and can therefore stably reconstitute the entire hematopoietic system following transplantation [1, 2]. This concept forms the basis for HSC transplantation (HSCT) therapies. These currently involve ablating a patient's dysfunctional hematopoietic system by radiation/chemotherapy and subsequent transplantation of healthy donor HSCs to reform a functional hematopoietic system within the patient.
Source: Experimental Hematology - Category: Hematology Authors: Source Type: research