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10/18/2016 - Medical Grand Rounds: Gene Therapy for Sickle Cell Disease: Scientific Fact or Science Fiction?





Credit Hours: CME 1.00

Target Audience:

Faculty, residents, fellows, and community physicians in General Internal Medicine and subspecialties.

Educational Objectives:

Upon completion of this activity, participants should be able to:

  • Increase patient treatment options with gene therapy.
  • Improve patient education with gene therapy information.
  • Improve diagnostic skills for recognizing high risk sickle cell disease.

Suggested Additional Reading:

  1. Goodman MA, Malik P. The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges. Ther Adv Hematol. 2016 Oct;7(5):302-315. Review. PubMed PMID: 27695619.
  2. The Lancet Haematology. Gene therapy for sickle cell disease. Lancet Haematol. 2016 Oct;3(10):e446. doi: 10.1016/S2352-3026(16)30130-2. PubMed PMID: 27692301.
  3. Traxler EA, Yao Y, Wang YD, Woodard KJ, Kurita R, Nakamura Y, Hughes JR, Hardison RC, Blobel GA, Li C, Weiss MJ. A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition. Nat Med. 2016 Sep;22(9):987-90. doi: 10.1038/nm.4170. Epub 2016 Aug 15. PubMed PMID: 27525524.

Brett Kaufman, PhD — Associate Professor of Medicine Division of Cardiology
No relationships with industry relevant to the content of this educational activity have been disclosed.
Greg Kato, MD — Professor of Medicine Division of Hematology/Oncology Director, Sickle Cell Center of Excellence
Dr. Kato receives Grant/Research support from Bayer AG. He is a consultant for Mast Therapeutics.
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.

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