Table 1.
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C9 K/O | Zebrafish | NA | NA | NA | No | No | Stable | No | NA | [34]; (Schmid, Hruscha, Haass, unpublished) |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