Regulation of gene expression in vivo following transduction by two separate rAAV vectors

KG Rendahl, SE Leff, GR Otten, SK Spratt, D Bohl… - Nature …, 1998 - nature.com
KG Rendahl, SE Leff, GR Otten, SK Spratt, D Bohl, M Van Roey, BA Donahue, LK Cohen…
Nature biotechnology, 1998nature.com
Control of gene expression is important to gene therapy for purposes of both dosing and
safety. In vivo regulation of gene expression was demonstrated following co-injection of two
separate recombinant adeno-associated virus vectors, one encoding an inducible murine
erythropoietin transgene and the other a transcriptional activator, directly into the skeletal
muscle of adult immunocompetent mice. Transcription was controlled by systemic
administration or withdrawal of tetracycline over an 18 week period, demonstrating that the …
Abstract
Control of gene expression is important to gene therapy for purposes of both dosing and safety. In vivo regulation of gene expression was demonstrated following co-injection of two separate recombinant adeno-associated virus vectors, one encoding an inducible murine erythropoietin transgene and the other a transcriptional activator, directly into the skeletal muscle of adult immunocompetent mice. Transcription was controlled by systemic administration or withdrawal of tetracycline over an 18 week period, demonstrating that the two vectors were capable of transducing the same cell. Cellular or humoral immune responses against the transactivator protein were not detected.
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