Doctors test 'Bubble Boy' gene cure with lower cancer risk

NCT ID NCT01410019

Summary

This trial tested a refined gene therapy to cure boys born with a severe, life-threatening immune deficiency called SCID-X1. For infants without a suitable bone marrow donor, doctors collected their own blood stem cells, inserted a corrected gene in the lab, and returned the cells. The main goal was to see if this safer version of the therapy could rebuild their immune systems without causing cancer.

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Contacts and locations

Locations

  • Hopital Necker

    Paris, 75015, France

Conditions

Explore the condition pages connected to this study.