The recent regulatory approval of the first haematopoietic stem and progenitor cell gene therapy (HSPC-GT) signals the start of a new era for gene therapy and highlights the potential contribution by high throughput cell culture technologies in propelling HSPC-GT from curing rare diseases to more common diseases.
Haematopoietic stem cells (HSCs) are the source of all the blood cells that circulate in our bodies throughout life. Arguably, no other cell types have a more profound and far-reaching influence on our well-being than HSCs. They reside in our bone marrow to continuously produce a variety of cells with vital tasks, such as the oxygenation of red blood cells, the termination of bleeding via platelets, and immunity via leukocytes that also provide immune defence to the central nervous system.
There is, however, a flipside to the pre-eminence of haematopoietic stem cells. When faulty HSCs emerge, devastating outcomes ensue, such as autoimmune diseases including multiple sclerosis and blood cancers like leukaemia. Thankfully, a solution to this situation is upon us: defective HSCs can be removed and replaced with healthy ones to tackle these life threatening indications by transplantation (HSCT) — a highly effective procedure pioneered by the late E.D. Thomas more than 50 years ago.
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