UCSF researcher Kyle Cromer, PhD, recently published a comprehensive new protocol that advances the field of genome editing for serious blood disorders such as sickle cell disease and thalassemia. His article in the August issue of STAR Protocols details how to achieve highly efficient CRISPR-based genome editing of hematopoietic stem cells — the stem cells that give rise to blood.
“This protocol allows scientists to take patient cells and correct the disease-causing mutation. These cells can then be re-transplanted into the patient to provide a lifelong cure,” explains Cromer, assistant professor of surgery in the UCSF School of Medicine and a member of UCSF’s Heath Innovation Via Engineering (HIVE) center.
The work is particularly important because the success of genome editing hinges on reaching sufficiently high editing rates in patient cells. In the recent paper titled Protocol for efficient CRISPR/AAV-mediated genome editing & erythroid differentiation of human hematopoietic stem & progenitor cells, Cromer’s team provides a step-by-step guide that enables researchers to achieve those clinically relevant levels — paving the way for more real-world treatments.
“Our protocol provides a critical foundation for researchers and clinicians striving to deliver safe, effective, and potentially curative therapies for patients living with devastating blood disorders,” he says.
Currently, he and his colleagues are adapting this strategy to enable highly efficient in vivo genome editing. “That would bypass the time- and cost-intensive process of isolating, editing, and re-transplanting patient cells,” Cromer says.