Appendix K. 
347 
Hacein-Bey-Abina A et a/., “Sustained correction of X- linked severe combined 
immunodeficiency by ex vivo gene therapy", New England Journal of Medicine 346, 
1185-1193: 18 Apr 2002. 
Aiuti A et al., "Correction of ADA-SCID by stem cell gene therapy combined vhth 
nonmyeloablative conditioning". Science 296, 2410-2413; 28 June 2002. 
Benedetti S et al., “Gene therapy of experimental brain tumors using neural 
progenitor cells". Nature Medicine 6, 447-450; Apr 2000. 
Jin, HK et ai; “Intracerebral transplantation of mesenchymal stem cells into acid 
sphingomyelinase-deficient mice delays the onset of neurological abnormalities and 
extends their life span": Journal of Clinical Investigation 109, 1183-1191; May 2002. 
Lee JY et ai., “Effect of bone morphogenetic protein-2-expressing muscle-derived 
cells on healing of critical-sized bone defects in mice". The Journal of Bone and Joint 
Surgery 83-A, 1032-1039; July 2001. 
Zeisberg M et ai., “BMP-7 counteracts TGF-Bl -induced epithelial-to-mesenchymal 
transition and reverses chronic renal injury ", Nature Medicine 7, 964-968; July 2003 
PRE -PUBLICATION VERSION 
