Sickle cell disease gene therapy education project

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

SC RED has partnered with NIH and the Democratizing Education Project to disseminate information relating to gene therapy. This was a project led by the National Human Genome Research Institute in partnership with other organizations and individuals, several of whom are on the SC RED team. The goal was to create patient educational materials that represented both scientific perspectives and patient voices, that did not come from any single clinical trial or company.
Gene therapy is an emerging field that holds promise for treating a wide range of genetic disorders, including sickle cell disease (SCD). SCD is caused by a mutation in the HBB gene, which codes for the beta-globin subunit of hemoglobin. Gene therapy for SCD involves introducing a functional copy of the HBB gene into the patient’s hematopoietic stem cells, which can then produce healthy red blood cells.
Fertility preservation is important for individuals with SCD who may require treatment exposing them to toxicity that can damage the ovaries or testes and affect fertility. Preserving reproductive cells or tissues before undergoing such treatments can enable individuals to have biological children in the future.
In the context of gene therapy, fertility preservation is particularly important because the process involves intensive chemotherapy or radiation to eliminate the patient’s existing bone marrow, which will be replaced with genetically modified stem cells. This can result in temporary or permanent infertility. Therefore, it is essential that individuals undergoing gene therapy for SCD have access to fertility preservation options, such as sperm or egg freezing, before undergoing treatment.
Inspired by the story of our CEO, SC RED is committed to ensuring fertility preservation is accessible to the community. Overall, the combination of gene therapy and fertility preservation can provide individuals with SCD the opportunity to both transform their disease and preserve their ability to have biological children in the future. YOU SHOULD NOT HAVE TO CHOOSE between transformative therapy or fertility. 

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

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 22, 2022

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About Us

Sickle Cell Reproductive Health Education Directive (SC RED) advocates for high quality sexual and reproductive health care for individuals living with all types of sickle cell disease

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