Hematopoietic stem cells (HSCs) have long been used to provide life-saving treatments for patients with malignant and non-malignant hematological disorders. While these treatments originally took the form of conventional bone marrow transplants, more recent breakthroughs in the field have provided cell therapy researchers with methods to correct genetic mutations in a patient’s own cells for autologous HSC transplantation.
We have compiled a selection of scientific resources to help you on your way in the complex and exciting field of hematopoietic cell therapy.
Description: In this webinar, Dr. Mark DeWitt will discuss the use of gene editing techniques for hematopoietic stem and progenitor cells HSPCs. Topics he will include are:
- Delivery of gene editing machinery to target cells
- Homology-directed repair for precise editing
- Culture conditions required for efficient editing
- Levels of correction needed for the treatment of monogenic disorders, such as sickle cell disease