Towards the genetic treatment of beta-thalassemia: new disease models, new vectors, new cells.
Moi
Paolo
P
Sadelain
Michel
M
D000818:Animals; D054337:Cell Dedifferentiation; D004195:Disease Models, Animal; D005347:Fibroblasts / Q000166:cytology; D017353:Gene Deletion; D005813:Genes, Synthetic; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000627:therapeutic use*; D005914:Globins / Q000235:genetics; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D051379:Mice; D008810:Mice, Inbred C57BL; D016688:Mice, Inbred NOD; D008817:Mice, Mutant Strains; D016513:Mice, SCID; D008822:Mice, Transgenic; D011993:Recombinant Fusion Proteins / Q000502:physiology; D033581:Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods; D019076:Transgenes; D017086:beta-Thalassemia / Q000235:genetics / Q000601:surgery / Q000628:therapy*
D011993:Recombinant Fusion Proteins; D005914:Globins