Condition group
Inherited red blood cell disorders
Conditions carried in the genes that change how red blood cells are shaped or how long they last.
Conditions in this group
Grouped by the part transplant usually plays. Treatment depends on the exact diagnosis, disease stage, prior treatment and the person’s health.
Cell or gene therapy options 2
Cell or gene therapies may be options for eligible patients in some countries. They do not all work the same way, use the same cells or replace the same type of transplant.
- In-depth guide
Beta thalassemia major (transfusion-dependent)
Transfusion-dependent beta-thalassemia is an inherited hemoglobin disorder that requires regular red-cell transfusions. Iron management is essential alongside transfusion. A donor transplant or an approved gene therapy may offer transfusion independence for selected people, but each requires careful assessment and long-term care.
Cell or gene therapy options - In-depth guide
Sickle cell disease
Sickle cell disease is a group of inherited hemoglobin disorders that can cause anemia, pain, infection and organ injury. Medicines, prevention and transfusion are central to care. A donor transplant or gene therapy can offer a major change in disease course for selected people, with substantial treatment risks.
Cell or gene therapy options
Limited transplant role 1
Other treatments are usual. Transplant may be considered in selected circumstances or research settings, as described on the condition page.
Other condition groups
Understanding transplant
Understanding can become action.
Some patients need a blood stem cell donor. Others receive different treatment. Wherever your interest began, you can help more people find the donor they need.
Join the registry
JBF points you to the official registry that serves your country. It explains who can join and what donation involves.
Help someone you love find a donor
If someone you love needs a donor, our family guide explains practical ways to help. A registration drive can add many potential donors at once, for them and for others.
Support this work
Gifts to the Jada Bascom Foundation support donor-awareness education like this page, community outreach, drive planning and referrals to official registries.
