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Gene Therapy
Gene Therapy with FBX-101 for Krabbe Disease
Recruiting1 awardPhase 1 & 2
Pittsburgh, Pennsylvania
This trial tests a gene therapy given through an IV after a stem cell transplant in patients with Krabbe disease. The therapy uses a virus to deliver healthy genes to help fix the genetic problem. Gene therapy using viral vectors has shown some success in extending survival in mouse models of Krabbe disease.
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