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Sickle cell disease gene therapy education project

It is imperative that patients with sickle cell disease who are considering a gene therapy clinical trial fully understand the meaning and limitations of an experimental treatment so they can make an informed decision on whether to participate. Patients need accessible, understandable and actionable educational materials to help them make such decisions.

About this project

The Democratizing Education for Sickle Cell Disease Gene Therapy project invited more than 50 stakeholders to participate in a series of workshops. These stakeholders represented the following groups: patients, parents of patients, health-professionals, community-based organizations, advocacy groups, industry leaders, government representatives and researchers.

 

The project group met over four days in both large and small group settings. Based on their expertise and role, participants were assigned to five educational focus areas:

  1. Understanding the red blood cell 
  2. Understanding  gene therapy approaches 
  3. Clinical trial process for sickle cell disease gene therapy
  4. Your mental health and sickle cell disease gene therapy participation
  5. Sickle cell disease gene therapy FAQs 

 

Each group determined key recommendations and presented that information to the larger group for further discussion and deliberation.

The Democratizing Education Project welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to DemocratizingEd@mail.nih.gov.

These educational materials are for informational purposes only. They are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use these educational materials to talk with your healthcare provider or a clinical trial team.

Patient education materials

Last updated: September 22, 2022