Table 1.

Mouse models useful for testing drug efficacy for potential SMA therapy

Model	Genotype	Survival (days)	Outcome Measures	References
Taiwanese	Smn−/−; SMN2(2Hung)+/± [Mice carry 1 or 2 copies of transgene]	Type I: ~10 Type II: ~14 Type III: Normal	Survival; motor function; tail and limb necrosis; motor neuron, NMJ, muscle and heart morphology and/or function	[38,48]
Line89	Smn−/−; SMN2(89Ahmb)+/+	5	Survival; motor function; motor neuron, NMJb and muscle morphology and/or function	[39]
Δ7 SMA	Smn−/−; SMN2(89Ahmb)+/+; SMNΔ7+/+	~14	Survival; motor function; motor neuron, NMJ, muscle and cardiac morphology and/or function	[40]
3 copy SMN2	Smn−/−; SMN2(N11); SMN2(N46)	14–16	Survival; motor function; motor neuron, NMJ and muscle morphology and/or function	[41]
F7 or exon 7 floxed	SmnF7/Δ7; NSE-Cre [Exon 7 loss in neurons]	25	Survival; motor function; motor neuron, NMJ and muscle morphology and/or function	[43]
F7 or exon 7 floxed	SmnF7/Δ7; HSA-Cre [Exon 7 loss in skeletal muscle]	33	Survival; motor function; motor neuron, NMJ and muscle morphology and/or function	[42]
2B	Smn2B/− [2B is defined by mutation in exon 7 splicing enhancer]	~30	Survival; motor function; neuromuscular junction and muscle morphology and/or function	[22]
SMNRT	Smn−/−; SMN2(89Ahmb) +/+; SMNΔ7RT +/+	34	Survival; motor function; motor neuron, NMJ and muscle morphology and/or function	[44]
Olig2-Cre	SmnF7/−; SMN2(89Ahmb)+/+; Olig2-Cre [Exon 7 loss in motor neuron progenitor cells]	365a	Motor function; motor neuron, NMJ and muscle morphology and/or function	[45]
Smn C>T	SmnC>T [SMN2 mutation inserted in mouse Smn]	Normal	Motor function; motor neuron, NMJ and muscle morphology and/or function	[46]
Allele C	SmnC/C [C is defined by a chimeric gene plus SMN2]	Normal	Ear, tail and limb necrosis; cardiac morphology and function	[47]

^a70% of these mice survived to 365 days

^bNMJ, neuromuscular junction