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In vivo retroviral gene transfer by direct intrafemoral injection results in correction of the SCID phenotype in Jak3 knock-out animals.
McCauslin CS, Wine J, Cheng L, Klarmann KD, Candotti F, Clausen PA, Spence SE, Keller JR. McCauslin CS, et al. Among authors: wine j. Blood. 2003 Aug 1;102(3):843-8. doi: 10.1182/blood-2002-12-3859. Epub 2003 Apr 10. Blood. 2003. PMID: 12689938 Free article.
Using this approach we have demonstrated in vivo retroviral gene transfer to colony-forming units-c (CFU-c), short-term reconstituting cells, and PHSCs. Moreover, direct intrafemoral injection of Jak3 knock-out mice with retroviral particles encoding the Jak3 gene resulted …
Using this approach we have demonstrated in vivo retroviral gene transfer to colony-forming units-c (CFU-c), short-term reconstitutin …