This clinical trial will access the toxicity and efficacy of infusion of gene modified cells
for patients with Fanconi anemia (FA). Infusion of autologous patient blood stem cells that
have been corrected in the laboratory by introduction of the normal gene may improve blood
counts in patients with FA.
Phase:
Phase 1
Details
Lead Sponsor:
Fred Hutchinson Cancer Research Center
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI) Rocket Pharma Limited