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Gulías, P. (Paola); Guerra-Varela, J. (Jorge); Gonzalez-Aparicio, M. (Manuela); et al. "Danio rerio as model organism for adenoviral vector evaluation". Genes. 10 (12), 2019, 1053
Viral vector use is wide-spread in the field of gene therapy, with new clinical trials starting
every year for different human pathologies and a growing number of agents being approved by
regulatory agencies. However, preclinical testing is long and expensive, especially during the early
stages of development. Nowadays, the model organism par excellence is the mouse (Mus musculus),
and there are few investigations in which alternative models are used. Here, we assess the possibility
of using zebrafish (Danio rerio) as an in vivo model for adenoviral vectors. We describe how
E1/E3-deleted adenoviral vectors achieve efficient transduction when they are administered to
zebrafish embryos via intracranial injection. In addition, helper-dependent (high-capacity) adenoviral
vectors allow sustained transgene expression in this organism. Taking into account the wide
repertoire of genetically modified zebrafish lines, the ethical aspects, and the affordability of this
model, we conclude that zebrafish could be an efficient alternative for the early-stage preclinical
evaluation of adenoviral vectors.