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Sickle cell disease gene therapy education project
Research Projects

The Big Picture

  • Sickle cell disease is an inherited disorder caused by a change in the gene that produces hemoglobin, the red blood cells that carry oxygen throughout the body.
  • In December 2023, the U.S. Food and Drug Administration (FDA) approved two gene therapies to treat sickle cell disease.
  • More than 50 scientists, physicians, patients and other experts worked together to develop content for the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in partnership with other organizations and individuals.
  • These education materials reflect the priorities and perspectives of both individuals living with sickle cell disease and the gene therapy and biomedical research communities. 
     

About this project

This project created patient-focused education materials for the sickle cell community. It is important that for patients and families who are considering gene therapy to understand the benefits and limitations of treatments so that they can make an informed decision on the therapies. Patients need accessible, understandable, and actionable educational materials to help them make such decisions.

To help navigate these new therapies, more than 50 scientists, physicians, patients and other experts worked together to develop content for the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in partnership with other organizations and individuals.

graphic showing people as part of community of stakeholders

Understanding the blood cell

Learn about the different types of hemoglobin.

Doctor and patient

Understanding gene therapy approaches

A variety of approaches target the blood stem cells to relieve or reduce your symptoms of sickle cell disease.

Doctor and patient consultation

Navigating sickle cell disease gene therapy

Gene therapy for sickle cell disease is available to you through FDA-approved therapies and through clinical trials.

Your mental health and gene therapy

Your mental and physical health are deeply connected. Mental health is important for your overall health and quality of life.

A couple in discussion

Sickle cell disease gene therapy FAQ

Discuss these questions with your doctor or health care provider.

family with a young girl reviewing forms with a healthcare worker

Patient resources for sickle cell disease gene therapy

Sickle cell disease resources for patients

a group of four healthcare workers seated at a table having a conversation

Resources for healthcare providers

Sickle cell disease resources for healthcare providers

Patient education videos

 
  • Patient education videos
     

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.

Last updated: September 12, 2024