University of Pittsburgh Health Sciences eLearning Environment Internet-based Studies in Education and Research
INTRODUCTION
6/8/2021 - Medical Grand Rounds: Bench to Bedside-Targeting Fetal Hemoglobin in Sickle Cell Disease & Clinical Application of Post-transcriptional Silencing of BCL11A
QUIZ
EVALUATION
CERTIFICATE
Credit Hours: CME 1.00
Faculty, residents, fellows, and community physicians in General Internal Medicine and subspecialties.
Targeting Fetal Hemoglobin in Sickle Cell Disease
Presented by: Martin Steinberg, MD
Clinical Application of Post-transcriptional Silencing of BCL11A
Presented by: David Williams, MD
Upon completion of this activity, participants should be able to:
- Describe the role of fetal hemoglobin as an inhibitor of sickle hemoglobin polymerization in sickle cell disease
- Review the varied pathophysiologies of sickle cell disease
- Compare old and new approaches to pharmacological induce fetal hemoglobin in sickle cell anemia
- Describe the relationship of fetal hemoglobin and sickle cell phenotype
- Explain the nature of BCL11A as target for treatment of sickle cell disease
- Review shmIR technology
Suggested additional reading:
- Buchanan Blood 2014
- Sankaran Nature 2009
- Esrick NEJM 2021
- Steinberg M.H., Thein S.L. Genetic Basis of Sickle Cell Disease, In, Sickle Cell Disease, Gladwin, M.T., Kato, G.J. Novelli, E.M. eds., McGraw Hill, 2021:11-32.
- Steinberg M.H. Fetal hemoglobin in sickle cell anemia. Blood 136: 2092, 2020. doi: 10.1182/blood.2020007645. PMID: 32808012
- Steinberg M.H. Fetal hemoglobin in sickle hemoglobinopathies: High HbF genotypes and phenotypes. J. Clin. Med. 2020. 9, 3782; doi:10.3390/jcm9113782. PMID: 33238542
Joint Accreditation Statement

The University of Pittsburgh School of Medicine designates this enduring material activity for a maximum of 1.0 AMA PRA Category 1 Credit[s]â„¢. Physicians should only claim credit commensurate with the extent of their participation in the activity. This educational activity is approved for 1.0 contact hours.
Other health care professionals will receive a certificate of attendance confirming the number of contact hours commensurate with the extent of participation in this activity.
Prior research funding Bluebird Bio for Research in Hemoglobinopathies. Co-founder of SAB and Orchard Therapeutics. Research funding from Orchard Therapeutics for X - SCID. Dr. Williams is a member of the Steering Committee for Novartis ETB115E2201. Insertion site analysis Advisory Board for Bluebird Bio & Biomarin. Co-Founder, Alerion Biosciences. Scientific Advisory Board for Beam Therapeutics. Chief Scientific Chair for Emerging Therapy Solutions. Scientific Advisory Board Geneception.
Dr. Steinberg is a consultant for Vertex/CRISPR Pharmaceuticals; Fulcrum Therapeutics; and Alexion Pharmaceuticals. He is an investor in Imara, and DMC.
This activity is approved for AMA PRA Category 1 Creditâ„¢
The University of Pittsburgh is an affirmative action, equal opportunity institution.