Nuria Morral

School of Medicine, Medical & Molecular Genetics

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High doses of a helper-dependent adenoviral vector yield supraphysiological levels of α₁-antitrypsin with negligible toxicity

Núria Morral; Robin J. Parks; Heshan Zhou; Claire Langston; Gudrun Schiedner; Janet Quinones; Frank L. Graham; Stefan Kochanek; Arthur L. Beaudet (Profiled Author: Nuria Morral)

Human Gene Therapy. 1998;9(18):2709-2716.

Optimal gene therapy for many disorders will require efficient transfer to cells in vivo, high-level and long-term expression, and tissue-specific regulation, all in the absence of significant toxicity or inflammatory responses. While recombinant adenoviral vectors are efficient for gene transfer to hepatocytes, their usefulness is limited by short duration of expression related, at least in part, to immune responses to viral proteins and by a low capacity for foreign DNA. A number of systems have been developed for producing adenoviral vectors devoid of all viral coding sequences. Using AdSTK109, a vector lacking all viral coding sequences and carrying the complete human α1-antitrypsin (hAAT) genomic DNA locus, we have demonstrated sustained expression for longer than 10 months in mice. Utilizing high doses of this vector for hepatic gene transfer in mice, we find that supraphysiological levels of hAAT can be achieved without hepatotoxicity.

PMID: 9874269    

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