Gene and Cell Therapies for Beta-Globinopathies

1. Clinical Features of β-Thalassemia and Sickle Cell Disease
Patrick T. McGann, Alecia C. Nero, Russell E. Ware

2. Genetic Basis and Genetic Modifiers of β-Thalassemia and Sickle Cell Disease
Swee Lay Thein

3. Current Standards of Care and Long Term Outcomes for Thalassemia and Sickle Cell Disease
Satheesh Chonat, Charles T. Quinn

4. Allogeneic/Matched Related Transplantation for β-Thalassemia and Sickle Cell Anemia
Françoise Bernaudin, Corinne Pondarré, Claire Galambrun, Isabelle Thuret

5. Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease
Courtney D. Fitzhugh, Allistair Abraham, Matthew M. Hsieh

6. Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia
Alisa C. Dong, Stefano Rivella

7. Reactivation of Fetal Hemoglobin for Treating β-Thalassemia and Sickle Cell Disease
Shuaiying Cui, James Douglas Engel

8. Genome Editing for the β-Hemoglobinopathies
Matthew H. Porteus

9. Gene and Cell Therapy for β-Thalassemia and Sickle Cell Disease with Induced Pluripotent Stem Cells (iPSCs): The Next Frontier
Eirini P. Papapetrou

Nyckelord: Biomedicine, Gene Therapy, Cell Biology