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Experience With Allogeneic Transplant for Sickle Cell Disease and the Prospects for Novel Gene Therapy Approaches

Dr. John Tisdale describes some of the efforts to bring curative therapies to sickle cell disease. He reviews why clinical trial participation is important for developing therapies, the current landscape for bone marrow transplantation strategies, and prospects for novel curative approaches. 

Educational objectives:

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

  • Describe the clinical trial participation is important for developing therapies for SCD
  • Recognize what the current landscape is for bone marrow transplantation strategies in SCD
  • Explain the prospects for novel curative gene therapy approaches based upon bone marrow transplantation

Reading Resources:

  1. Measurements of red cell deformability and hydration reflect HbF and HbA2 in blood from patients with sickle cell anemia.
  2. Parrow NL, Tu H, Nichols J, Violet PC, Pittman CA, Fitzhugh C, Fleming RE, Mohandas N, Tisdale JF, Levine M.
  3. Blood Cells Mol Dis. 2017 Apr 18;65:41-50. doi: 10.1016/j.bcmd.2017.04.005.
  4. Bone marrow as a hematopoietic stem cell source for gene therapy in sickle cell disease (SCD): evidence from rhesus and SCD patients.
  5. Uchida N, Fujita A, Hsieh MM, Bonifacino A, Krouse AE, Metzger ME, Donahue RE, Tisdale JF. Hum Gene Ther Clin Dev. 2017 Apr 27. doi: 10.1089/humc.2017.029.
  6. RNA Trans-Splicing Targeting Endogenous β-Globin Pre-Messenger RNA in Human Erythroid Cells.Uchida N, Washington KN, Mozer B, Platner C, Ballantine J, Skala LP. Raines L, Shvygin A, Hsieh MM, Mitchell LG, Tisdale JF.
  7. Hum Gene Ther Methods. 2017 Apr;28(2):91-99. doi: 10.1089/hgtb.2016.077. Epub 2017 Feb 14.

Disclosures:

Dr. Tisdale has reported no relevant relationships with proprietary entities producing health care goods or services.

All presenters disclosure of relevant financial relationships with any entity producing, marketing, re-selling, or distributing health care goods or services, used on, or consumed by, patients is listed above.  No other planners, 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 to disclose.

Accreditation Statement:

The University of Pittsburgh School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

The University of Pittsburgh School of Medicine designates this enduring material for a maximum of 1.25 AMA PRA Category 1 Credits™. Each physician should only claim credit commensurate with the extent of their participation in the activity. Other health care professionals are awarded (0.125) continuing education units (CEU) which are equivalent to 1.25 contact hour.

For your credit transcript, please access our website 4 weeks post-completion at http://ccehs.upmc.com and follow the link to the Credit Transcript page. If you do not provide the last 5 digits of your SSN on the next page you will not be able to access a CME credit transcript. Providing your SSN is voluntary.

ABIM MOC Part 2 Credit

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.25 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit. 

To receive your ABIM MOC Part 2 credit, you will need to complete the post-test and evaluation process and provided your date of birth and ABIM number, along with the other required fields.

Release Date: 10/19/2017 | Last Modified On: 10/19/2017 | Expires: 6/24/2020

Presenter

John F. Tisdale, MD

John F. Tisdale, MD Senior Investigator
Molecular and Clinical Hematology Section
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