AUTOSOMAL RECESSIVE

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Scientists test One-Time gene fix for 'Bubble Boy' disease

Disease control ENROLLING_BY_INVITATION

This study is testing a new gene therapy for people with a rare, inherited immune disorder called p47 chronic granulomatous disease (CGD). Doctors will collect a patient's own blood stem cells, add a corrected copy of the missing gene in a lab, and then return the modified cells …

Matched conditions: AUTOSOMAL RECESSIVE

Phase: PHASE1, PHASE2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control

Last updated Apr 01, 2026 23:11 UTC

AUTOSOMAL RECESSIVE

Get alerted when new AUTOSOMAL RECESSIVE trials appear

Scientists test One-Time gene fix for 'Bubble Boy' disease