title: Phase I/II Gene Therapy Trial of Fanconi Anemia Patients with a New Orphan Drug Consisting of a Lentiviral Vector Carrying the FANCA Gene: A Coordinated International Action (EuroFancolen).
mesh_terms: D000818:Animals; D001596:Benzylamines; D000080027:Cyclams; D005199:Fanconi Anemia / Q000235:genetics / Q000628:therapy*; D052217:Fanconi Anemia Complementation Group A Protein / Q000235:genetics*; D005822:Genetic Vectors; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D019650:Hematopoietic Stem Cell Mobilization; D006571:Heterocyclic Compounds / Q000627:therapeutic use; D006801:Humans; D007391:International Cooperation; D016086:Lentivirus / Q000235:genetics*; D018345:Mice, Knockout; D009965:Orphan Drug Production
chemical_list: D001596:Benzylamines; D000080027:Cyclams; D052217:Fanconi Anemia Complementation Group A Protein; D006571:Heterocyclic Compounds; D016179:Granulocyte Colony-Stimulating Factor; C088327:plerixafor