Gene therapy in sickle cell disease: Attitudes and informational needs of patients and caregivers

Abstract excerpt

BACKGROUND: Sickle cell disease (SCD) is an inherited blood disorder that results in serious morbidity and early mortality. Novel therapies for SCD, most notably genetic therapies (GTs) and HLA-mismatched donor hematopoietic cell transplantation, are in clinical trials. While potentially curative, these interventions are some of the most intensive treatments for SCD and are associated with serious and...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to CRISPR and Genetic Engineering
• Share to Hemoglobinopathies and Related Disorders
• Share to Virus-based gene therapy research