Doctors track patients for 15 years after pioneering gene therapy for devastating immune disease
NCT ID NCT06282432
Summary
This study is tracking 9 patients for up to 15 years after they received an experimental gene therapy for a rare and severe immune disorder called LAD-I. The therapy involved modifying a patient's own blood stem cells to correct the genetic defect. Researchers want to see if the treatment remains safe and effective long-term, and if it helps patients avoid infections and other serious complications.
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Contacts and locations
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Locations
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Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
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University College London Great Ormond Street Institute of Child Health (GOSH)
London, WC1N 1EH, United Kingdom
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095-1489, United States
Conditions
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