Evaluation and application of modularly assembled zinc-finger nucleases in zebrafish

C Zhu, T Smith, J McNulty, AL Rayla… - …, 2011 - journals.biologists.com
C Zhu, T Smith, J McNulty, AL Rayla, A Lakshmanan, AF Siekmann, M Buffardi, X Meng
Development, 2011journals.biologists.com
Zinc-finger nucleases (ZFNs) allow targeted gene inactivation in a wide range of model
organisms. However, construction of target-specific ZFNs is technically challenging. Here,
we evaluate a straightforward modular assembly-based approach for ZFN construction and
gene inactivation in zebrafish. From an archive of 27 different zinc-finger modules, we
assembled more than 70 different zinc-finger cassettes and evaluated their specificity using
a bacterial one-hybrid assay. In parallel, we constructed ZFNs from these cassettes and …
Zinc-finger nucleases (ZFNs) allow targeted gene inactivation in a wide range of model organisms. However, construction of target-specific ZFNs is technically challenging. Here, we evaluate a straightforward modular assembly-based approach for ZFN construction and gene inactivation in zebrafish. From an archive of 27 different zinc-finger modules, we assembled more than 70 different zinc-finger cassettes and evaluated their specificity using a bacterial one-hybrid assay. In parallel, we constructed ZFNs from these cassettes and tested their ability to induce lesions in zebrafish embryos. We found that the majority of zinc-finger proteins assembled from these modules have favorable specificities and nearly one-third of modular ZFNs generated lesions at their targets in the zebrafish genome. To facilitate the application of ZFNs within the zebrafish community we constructed a public database of sites in the zebrafish genome that can be targeted using this archive. Importantly, we generated new germline mutations in eight different genes, confirming that this is a viable platform for heritable gene inactivation in vertebrates. Characterization of one of these mutants, gata2a, revealed an unexpected role for this transcription factor in vascular development. This work provides a resource to allow targeted germline gene inactivation in zebrafish and highlights the benefit of a definitive reverse genetic strategy to reveal gene function.
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