Gene therapy aims to fix bone marrow failure in fanconi anemia
NCT ID NCT03351868
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This trial tests a gene therapy for Fanconi anemia, a rare genetic disorder that causes bone marrow failure and increases cancer risk. Participants receive their own stem cells that have been genetically corrected with a lentiviral vector to fix the faulty FANCA gene. The study evaluates safety and whether the treatment can improve blood cell counts and reduce disease complications.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
gene-modified stem cells
What this could lead to
If successful, this gene therapy could restore normal blood cell production and reduce the need for transfusions or bone marrow transplants in people with Fanconi anemia.
What could go wrong
This is an early-phase trial with only 10 participants, so results may not apply to everyone. Risks include immune reactions or the gene therapy not working as expected.
Disclaimer
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This is a summary of
the original study
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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.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••