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6/8/2021 - Medical Grand Rounds: Bench to Bedside-Targeting Fetal Hemoglobin in Sickle Cell Disease & Clinical Application of Post-transcriptional Silencing of BCL11A





Credit Hours: CME 1.00

Target Audience:

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

Educational Objectives:

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 & Joint Accreditation Statement - Note: This Accreditation Statement Supersedes All Other Statements:

Suggested additional reading:

  1. Buchanan Blood 2014
  2. Sankaran Nature 2009
  3. Esrick NEJM 2021
  4. 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.
  5. Steinberg M.H. Fetal hemoglobin in sickle cell anemia. Blood 136: 2092, 2020. doi: 10.1182/blood.2020007645. PMID: 32808012
  6. 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
In support of improving patient care, the University of Pittsburgh is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME) and the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

The University of Pittsburgh School of Medicine designates this enduring material activity for a maximum of 1.0 AMA PRA Category 1 Credit[s]™. 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.

David Williams, MD — Chief of Hematology/Onocology, Boston Children's Hospital, Lealand Fikes Chair of Pediatrics, Harvard Medical School
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.
Martin Steinberg, MD — Professor of Medicine, Pediatrics, Pathology and Laboratory Medicine Boston University School of Medicine
Dr. Steinberg is a consultant for Vertex/CRISPR Pharmaceuticals; Fulcrum Therapeutics; and Alexion Pharmaceuticals. He is an investor in Imara, and DMC.
No other members of the planning committee, speakers, presenters, authors, content reviewers and/or anyone else in a position to control the content of this education activity have relevant financial relationships with any companies whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.

This activity is approved for AMA PRA Category 1 Credit™

The University of Pittsburgh is an affirmative action, equal opportunity institution.