Núria Morral

TL;DR: Using an adenoviral vector with all viral coding sequences deleted and containing the complete human α1-antitrypsin (PI) locus, intravenous injection in mice resulted in high levels of very stable expression for more than ten months and decreased acute and chronic toxicity.

TL;DR: The duration of expression of AdSTK109 in the liver of baboons and compared it to first-generation vectors expressing hAAT are examined to suggest that long-term expression of transgenes should be possible by combining the reduced immunogenicity and toxicity of helper-dependent vectors with sequential delivery of vectors of different serotypes.

TL;DR: Haplotype data demonstrate that ΔF508 occurred more than 52,000 years ago, in a population genetically distinct from any present European group, and spread throughout Europe in chronologically distinct expansions, which are responsible for the different frequencies of ΔF50 in Europe.

TL;DR: Using AdSTK109, a vector lacking all viral coding sequences and carrying the complete human alpha1-antitrypsin genomic DNA locus, it is found that supraphysiological levels of hAAT can be achieved without hepatotoxicity in mice.

TL;DR: It is indicated that persistence of expression of hAAT can be markedly decreased by administration of high doses of vector in a manner consistent with a nonimmune-mediated toxicity following injection and host immune responses to the reporter can limit duration of expression.