XSCID

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Groundbreaking gene editing trial aims to fix immune system in 'Bubble Boy' disease

Disease control ENROLLING_BY_INVITATION

This study tests a new gene therapy for X-linked severe combined immunodeficiency (X-SCID), a rare disorder that leaves people without a working immune system. Researchers take a person's own blood stem cells, fix the faulty gene using a precise editing technique called base edit…

Matched conditions: XSCID

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

Last updated May 15, 2026 11:53 UTC

XSCID

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Groundbreaking gene editing trial aims to fix immune system in 'Bubble Boy' disease