[A study of gene therapy for beta-thalassemia. I. Retroviral-mediated transfer of human beta-globin gene into Friend cells].
We used a replication-defective retrovirus vector containing the human beta-globin gene to transfect the ecotropic packaging cell line psi-2, and then harvested the virus supernatant to infect the amphotropic packaging cell line PA317. As a result, a recombinant vector producing cell line (PIW beta) with a titer of 1.1 x 10(5) CFU/ml was isolated. By infecting Friend cells with this high titer virus vector, high efficiency gene transfer was achieved. Southern blot hybridization analysis indicated that the intact human beta-globin gene was stably integrated into the genome of the target cells. Our results provide a useful reference for further studies of gene therapy of beta-thalassemia.
Shu
H
H
Institute of Basic Medical Sciences, Beijing.
D015139:Blotting, Southern; D002460:Cell Line; D004274:DNA, Recombinant; D005622:Friend murine leukemia virus; D015316:Genetic Therapy; D005822:Genetic Vectors; D005914:Globins / Q000235:genetics*; D006801:Humans; D004915:Leukemia, Erythroblastic, Acute / Q000473:pathology; D012190:Retroviridae / Q000235:genetics*; D013789:Thalassemia / Q000628:therapy*; D014162:Transfection
D004274:DNA, Recombinant; D005914:Globins