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Table 1 Comparison of Stable C9orf72 stable mouse and zebrafish mutants showing the distinct phenotype and utility of each model

From: Stable transgenic C9orf72 zebrafish model key aspects of the ALS/FTD phenotype and reveal novel pathological features

DNA Construct Species RNA Foci Sense RAN DPR Antisense RAN DPR ALS phenotype FTD like phenotype Stable or Transient Phenotype amenable to drug screen Efficacy of riluzole Reference
BAC HRE Mouse Yes Yes No Yes Yes Stable No Not tested [14]
BAC HRE Mouse Yes Yes No Yes Yes Stable No Not tested [20]
BAC HRE Mouse Yes Yes No No No Stable No Not tested [29]
BAC HRE Mouse Yes Yes No No No Stable No Not tested [30]
C9 K/O Mouse NA NA NA No No Stable No NA [1, 13, 17, 35]
C9 K/D Zebrafish NA NA NA Axonal growth defect No Transient No Not tested [6]
C9 K/O Zebrafish NA NA NA No No Stable No NA [34]; (Schmid, Hruscha, Haass, unpublished)
C9 HRE Zebrafish Yes No No No (Mild cardiac phenotype) No Stable No Not tested [28]
C9-ATG GA Zebrafish Yes No No No (Severe cardiac phenotype) No Stable No (lethal) Not tested [28]
C9 -ATG HRE Zebrafish Yes Yes Yes Yes Yes Stable Yes Yes Current manuscript
  1. NA Not applicable, K/O Knockout, K/D Knockdown, Grey boxes represents the features that represent similarity to human ALS/FTD or utility in the high throughput screening of novel therapeutics