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Evidence: AAV-based gene therapies deliver a gene directly into the body inside a viral capsid; the immune system typically forms neutralizing antibodies to that capsid after the first dose, which blocks a second dose of the same product. Live-cel works ex vivo: a lentiviral vector corrects the patient's own stem cells in the lab, and only the corrected cells are returned.
Interpretation: Because no viral capsid is given to the patient, Live-cel is designed to keep a re-dosing path open if enzyme levels decline over time. That could matter for patients and for long-term value.
Uncertainty: Re-dosing has not been tested in people. Whether and when a second treatment would be needed, and how well it would work, are open questions for future studies.
Questions about re-dosing? Post them below and we'll answer here.
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