Table 1.

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

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C9 K/O	Zebrafish	NA	NA	NA	No	No	Stable	No	NA	[34]; (Schmid, Hruscha, Haass, unpublished)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

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