Advances in Gene Therapy for Sickle Cell Disease and Thalassemia
This activity consists of a recorded presentation from the "Essentials of Oncology, Solid Organ and Blood/Marrow Transplant Management for the Health Care Team" conference held in Scottsdale, Ariz., March 14–15, 2022.
Most treatment options for sickle cell disease (SCD) and beta (β)-thalassemia — both caused by mutations that affect hemoglobin β-globin chain function — are crises-focused, pain-focused, or maintenance-oriented. While allogeneic hematopoietic stem cell transplantation (allo-HSCT) is curative for both diseases, difficulties and risks surround the procedure, including problems in donor matching and graft-versus-host disease. Several new developments in gene therapy are now offering hope for a cure for SCD and β-thalassemia. This session will examine the genetic approaches being explored for these diseases, such as the introduction of a corrected or modified β-globin gene and gene-sequence editing. It will also explore the genetic modalities utilized, such as genome-editing technologies including the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene-editing system and lentiviral vectors. The safety, efficacy and challenges associated with these therapies will also be outlined.
This activity is provided by OptumHealth Education.
There was no commercial support for this activity.
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This activity is designed to meet the educational needs of case managers, nurses, pharmacists, pharmacy technicians, physicians, psychologists, social workers, dietitians, and other health care professionals who are interested in gene therapy, sickle cell disease and β-thalassemia.
At the end of this educational activity, participants should be able to:
- Identify the genetic mutations behind the pathogenesis of SCD and β-thalassemia.
- Describe the gene-editing modalities through which SCD and β -thalassemia gene corrections are being attempted.
- Discuss the goals of these potential therapies regarding altering gene expression and resulting clinical presentations of disease.
- Define the potential safety and efficacy profiles of these gene therapies based on data from current studies.
Stephan A. Grupp, MD, PhD
Section Chief, Cellular Therapy and Transplant Section
Director, Cancer Immunotherapy Program
Medical Director, Cell and Gene Therapy Laboratory
Children's Hospital of Philadelphia
About the presenter
Dr. Stephan A. Grupp is section chief of the cellular therapy and transplant section, director of the Cancer Immunotherapy Program, and medical director of the Cell and Gene Therapy Laboratory. He holds the Yetta Deitch Novotny Endowed Chair in pediatric oncology.
As an attending physician in the Cancer Center, Dr. Grupp takes on many roles at CHOP. He received an MD and PhD from the University of Cincinnati College of Medicine and completed residency and fellowships at Harvard Medical School, Boston Children’s Hospital, Dana Farber Cancer Institute and Brigham and Women’s Hospital, before coming to CHOP in 1996.
In one of his clinical roles, he works with patients with the most aggressive form of neuroblastoma, a difficult-to-treat childhood cancer that begins in the peripheral (non-brain) nerve tissue of infants and young children. He works alongside a world-class team of physicians and multidisciplinary specialists who are dedicated to treating this disease. The tandem-transplant approach that is now the standard of care across the country was developed by this group at CHOP. In addition, the neuroblastoma team at CHOP does studies of the patient’s genetics and the unique characteristics of their disease to offer a personalized treatment approach. The team also did the nationwide clinical trial establishing antibody-based immunotherapy as the new standard of care in neuroblastoma.
Elizabeth Albert, MD
Clinical Activity Manager
Eden Prairie, MN
Matthew Cooper, MD
President, UNOS Board of Directors;
Director, Kidney and Pancreas Transplantation
Medstar Georgetown Transplant Institute;
Professor of Surgery
Georgetown University School of Medicine
Rebecca Gleason, RN, CCM
Eden Prairie, MN
Alan N. Langnas, DO, FACS
Professor of Surgery
Director, Center of Transplantation
Chief, Section of Transplantation,
Department of Surgery
University of Nebraska Medical Center;
Director of Liver Transplantation
C. Fred LeMaistre, MD
Senior Vice President
Senior Vice President, Market Operations
Tina Rydland, PharmD
Disclosures of relevant financial relationships
In accordance with the Accreditation Council for Continuing Medical Education's (ACCME) Standards for Integrity and Independence in Accredited Continuing Education, OptumHealth Education (OHE) requires all those in control of educational content to disclose their financial relationships with ineligible companies within the prior 24 months. Ineligible companies are defined by the ACCME as companies whose primary business is producing, marketing, selling, re-selling, or distributing health care products used by or on patients. Individuals must disclose all financial relationships, regardless of the amount, with ineligible companies and regardless of their view of the relevance of the relationship to the education. OHE ensures that the content is independent of commercial bias.
Dr. Grupp has indicated that he is a consultant for Novartis and Vertex, serves on the advisory board for Novartis and Vertex, and receives grant/research funding from Novartis and Vertex.
The activity planners have no financial relationships to disclose.
Method for calculating CE credit
CE credit was calculated by the complexity of content.
In support of improving patient care, OptumHealth Education is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC) to provide continuing education for the health care team.
Credit designation statements
This activity was planned by and for the health care team, and learners will receive 0.75 Interprofessional Continuing Education (IPCE) credits for learning and change.
The participant will be awarded up to 0.75 contact hour(s) of credit for attendance and completion of supplemental materials.
The American Academy of Nurse Practitioners Certification Program (AANPCP) accepts credit from organizations accredited by the ACCME and ANCC.
This activity is approved for 0.75 contact hour(s) ([0.075] CEU) in states that recognize ACPE.
Attending the full program will earn 0.75 contact hour(s).
Unique Activity Number(s): JA0007123-0000-22-151-H01-P/T
OptumHealth Education designates this enduring activity for a maximum of 0.75 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
The American Academy of Physician Assistants (AAPA) accepts credit from organizations accredited by the ACCME.
The Commission for Case Manager Certification has approved this program for a maximum of 0.75 clock hour(s) for Certified Case Managers (CCM).
Completion of this RD/DTR profession-specific or IPCE activity awards CPEUs (One IPCE credit=One CPEU).
If the activity is dietetics-related but not targeted to RDs or DTRs, CPEUs may be claimed which are commensurate with participation in contact hours (One 60 minute hour=1 CPEU).
RD's and DTRs are to select activity type 102 in their Activity Log. Performance indicator selection is at the learner's discretion.
Psychologists and social workers
Participants must have attended the entire Web-based activity and completed an evaluation to receive a certificate. Partial credit is not available.
OptumHealth Education is approved by the American Psychological Association (APA) to offer continuing education for psychologists. OptumHealth Education maintains responsibility for this program. 0.75 CE hour.
As a Jointly Accredited Organization, OptumHealth Education is approved to offer social work continuing education by the Association of Social Work Boards (ASWB) Approved Continuing Education (ACE) program. Organizations, not individual courses, are approved under this program. State and provincial regulatory boards have the final authority to determine whether an individual course may be accepted for continuing education credit. OptumHealth Education maintains responsibility for this course. Social workers completing this course receive 0.75 enduring continuing education credit.
A certificate of attendance will be provided to learners upon completion of activity requirements, enabling participants to register with licensing boards or associations that have not been preapproved for credits. To apply for credit types not listed above, participants should use the procedure established by the specific organization with which they wish to obtain credit.
- 0.75 ACPE - Pharmacists
- 0.75 ACPE - Pharmacy Technicians
- 0.75 AMA - Physicians
- 0.75 ANCC - Nurses
- 0.75 APA - Psychologists
- 0.75 Attendance - General Attendance
- 0.75 CCMC - General - Case Managers
- 0.75 CDR - Dietitians
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