Species [Refs.] | Genetic anomaly | Modification approach | Pathology and phenotypes |
---|---|---|---|
TDP-43 (M337V) | Brain regional expression of transgenic TDP-43 | Cytoplasmic accumulation of mutant TDP-43, motor function deficits | |
Monkey [210] | TDP-43 (WT) | Spinal cord expression of human WT TDP-43 | Progressive motor weakness and muscle atrophy. Cytoplasmic mislocalization of TDP-43 |
SOD1 | Intrathecal delivery of AAV encoding SOD1-specific microRNA | Not described | |
Monkey [213] | FUS | Injection of FUS-targeted shRNA | Not described |
Pig [204] | TDP-43 (M337V) | Embryonic expression of transgenic TDP-43 | Severe phenotypes and early death |
Pig [202] | hSOD1 (G93A) | Embryonic expression of transgenic SOD1 | Hind limb movement deficits, loss of motor neurons, formation of neuronal intranuclear inclusions in early disease stage |
Pig [201] | hSOD1 (G93A) | Embryonic expression of transgenic SOD1 | No ALS-like phenotypes |
SOD1 (T18S, E40K) | Spontaneous | Progressive axonal degeneration, muscle atrophy, and SOD1 inclusions |