Gene fix for thalassemia: first human test shows promise
NCT ID NCT01639690
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study tests a gene therapy for adults with β-thalassemia major, a severe blood disorder requiring frequent transfusions. Doctors take the patient's own stem cells, fix the faulty gene, and return the corrected cells via an IV. A low dose of busulfan prepares the body to accept the new cells. The main goals are to check safety and see if the treated cells can produce normal red blood cells.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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Locations
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Hospital "V.Cervello" Uoc Ematologiaii E Malattie Rare
Palermo, 90146, Italy