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• W1138747448 abstract "Abstract Although zebrafish is used to model human diseases through mutational and morpholino-based knockdown approaches, there are currently no robust transgenic knockdown tools. Here we investigate the knockdown efficiency of three synthetic miRNA-expressing backbones and show that these constructs can downregulate a sensor transgene with different degrees of potency. Using this approach, we reproduce spinal muscular atrophy (SMA) in zebrafish by targeting the smn1 gene. We also generate different transgenic lines, with severity and age of onset correlated to the level of smn1 inhibition, recapitulating for the first time the different forms of SMA in zebrafish. These lines are proof-of-concept that miRNA-based approaches can be used to generate potent heritable gene knockdown in zebrafish." @default.
• W1138747448 created "2016-06-24" @default.
• W1138747448 creator A5044978602 @default.
• W1138747448 creator A5055959126 @default.
• W1138747448 creator A5065354759 @default.
• W1138747448 date "2015-06-08" @default.
• W1138747448 modified "2023-10-07" @default.
• W1138747448 title "Effective heritable gene knockdown in zebrafish using synthetic microRNAs" @default.
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• W1138747448 doi "https://doi.org/10.1038/ncomms8378" @default.
• W1138747448 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/4468906" @default.
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