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INTRODUCTION

ABIM MOC Activity ID

10/2/2018 - Medical Grand Rounds: Modified CRISPR/Cas for Safe and Effective Sickle Gene Correction in Patient Blood Stem Cells

QUIZ

EVALUATION

CERTIFICATE

INTRODUCTION

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:

  • Appraise patient outcomes using current therapies for sickle cell disease.
  • Outline diagnostic skills for identifying sickle cell disease patients at high risk for morbidity and mortality.
  • Examine patient outcomes through knowledge of current and future potential to refer patients for gene therapy clinical research.

Suggested Additional Reading:

  1. Li C, Ding L, Sun CW, Wu LC, Zhou D, Pawlik KM, Khodadadi-Jamayran A, Westin E, Goldman FD, Townes TM. Novel HDAd/EBV Reprogramming Vector and Highly Efficient Ad/CRISPR-Cas Sickle Cell Disease Gene Correction. Sci Rep. 2016 Jul 27;6:30422. doi: 10.1038/srep30422. PubMed PMID: 27460639; PubMed Central PMCID: PMC4961958.
  2. Chang CW, Lai YS, Westin E, Khodadadi-Jamayran A, Pawlik KM, Lamb LS Jr, Goldman FD, Townes TM. Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting. Cell Rep. 2015 Sep 8;12(10):1668-77. doi: 10.1016/j.celrep.2015.08.013. Epub 2015 Aug 28. PubMed PMID: 26321643.
  3. Kato GJ, Piel FB, Reid CD, Gaston MH, Ohene-Frempong K, Krishnamurti L, Smith WR, Panepinto JA, Weatherall DJ, Costa FF, Vichinsky EP. Sickle cell disease. Nat Rev Dis Primers. 2018 Mar 15;4:18010. doi: 10.1038/nrdp.2018.10. Review. PubMed PMID: 29542687.
  4. Jayavaradhan R, Malik P. Genetic Therapies for Sickle Cell Disease. Pediatr Clin North Am. 2018 Jun;65(3):465-480. doi: 10.1016/j.pcl.2018.01.008. Review. PubMed PMID: 29803277.

Authors:
Gregory Kato, MD — Professor of Medicine, director of the Adult Sickle Cell Center of Excellence for the UPP Division of Hematology-Oncology, and director of the Vascular Medicine Institute/Institute for Transfusion Medicine Sickle Cell Center of Excellence
Dr. Kato receives research support from Bayer. Dr. Kato’s past research support was from AesRx. Dr. Kato is a consult for Bioverativ, Novartis, Global Blood Therapeutics.
Tim M. Townes, PhD — Professor Emeritus Department of Biochemistry and Molecular Genetics Schools of Medicine and Dentistry University of Alabama at Birmingham
Dr. Townes is a stockholder with HemEdits, LLC
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.