Sickle Cell Disease | CHOP Research Institute

Sickle Cell Disease

Published on
Sep 30, 2022
Learn about novel findings and new grants from Children’s Hospital of Philadelphia researchers.

Dr. Thompson is the chief of the Division of Hematology at CHOP and holds the Elias Schwartz, MD, Endowed Chair in Hematology. Her research focuses on sickle cell disease and thalassemia.

thompsona7 [at]
Published on
Jun 24, 2022
This week’s In the News features endocrinology research, news on gene therapy in blood disorders, CAR T-cell therapy, and more.

This Phase 2 clinical research study is evaluating the safety and effectiveness of a once-daily oral investigational medicine that may improve anemia and reduce the need for red blood cell (RBC) transfusions among some patients who are chronically transfused.

 A gene in stem cells will then be edited to help the stem cells make more fetal hemoglobin. Higher levels of fetal hemoglobin can improve red blood cell sickling.

Published on
Dec 14, 2021
A new Diversity Fellow, Dr. Prussien studies barriers and facilitators of transition to adult medical care in adolescents with SCD.

The purpose of this research study is to compare transplant (HCT/BMT) to standard of care treatment in young adults with SCD who have experienced complications.

Exploring the possibilities for treating disorders during fetal development.

Dr. Chou's research laboratory focuses on regenerative blood cellular therapy, the mechanisms of normal human hematopoietic development, and how these become disrupted in hematologic diseases.

The purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 12 to 35 years who have been diagnosed with severe sickle cell disease.