Hemoglobinopathies and Cellular Therapy: What Pediatricians Need to Know About Indications and Counseling.
The pathophysiology, diagnosis, management, and curative options for SCD and transfusion-dependent thalassemia are summarized, emphasizing allogeneic hematopoietic stem cell transplantation and approved gene therapies.
Obadah Alquadan, Rasha Deebaja, Avanti Gupte et al.
· Pediatric annals · 1 citation