Gene editing could cure sickle cell by boosting fetal hemoglobin
NCT ID NCT07708350
First seen Jul 16, 2026 · Last updated Jul 17, 2026 · Updated 1 time
Summary
This pilot trial tests a gene-editing approach for sickle cell disease. Participants receive their own blood stem cells that have been edited to increase fetal hemoglobin, a healthy type of hemoglobin that prevents sickling. The goal is to cure the disease with a one-time treatment, similar to a bone marrow transplant but using the patient's own cells. The trial enrolls 5 people aged 13-40 with severe sickle cell disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- a person's own blood stem cells edited with a gene-editing tool to boost fetal hemoglobin
- What this could lead to
- If successful, this approach could provide a one-time cure for sickle cell disease by enabling the body to produce healthy fetal hemoglobin.
- What could go wrong
- This is a very early, small pilot trial with only 5 participants, so results may not apply broadly. Risks include chemotherapy side effects and the possibility that the edited cells do not engraft or produce enough fetal hemoglobin.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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