Table 1.

Summary of SOD1 silencing with RNAi

Reference	Injection site	Dose	Vector	% of transduction	SOD1 protein reduction	Extension in survival
Ralph et al. [24]	Intramuscular (hindlimb, facial, tongue, diaphragm and intercostals)	120 μl (7 × 108–1 × 109 transducing units/ml)	Lentiviral vectors based on the equine infectious anemia virus	95% in vitro	70% in vitro	227 days
Raoule et al. [25]	Bilaterally in the lumbar L3–L4 region	3 μl (60,000 ng of p24 antigen/ml)	Self-inactivating lentiviral vectors	20% in vivo	70% in vitro	–
					52% in vivo
Miller et al. [28]	Intra-muscular (right lower hind limb)	25 μl (1 × 1012 particles/ml)	AAV2-siRNA-GFP	–	–	–
Miller et al. [27]	Intra-muscular (hindlimbs)	1 × 1011 AAV2	AAV2-siRNA	–	60% in vivo	–
		1 × 107 lentiviral particles	Lentiviral siRNA
					60% in vivo
Towne et al. [30]	Intramuscular (gastrocnemius) intravenous	30 μl (2 × 109 vg)	AAV6 shSOD1	3.9% in vivo	59% in vivo	No effect
		200 μl (2 × 1011 vg)		–	–	–
Rizvanov et al. [32]	Lumbar spinal cord (sciatic nerve)	–	No vectors	–	–	–
Wu et al. [33]	Intraspinal cord (sciatic nerve)	7 μl (1.25 × 109 viral particles)	RAd-shR–SOD1-EGFP	–	–	10 days
		7 μl (5 × 108 viral particles)	AAV2-U6–shR-SOD1-EGFP			No effect
Towne et al. [37]	Intramuscular (hindlimb, forelimb, intercostal, facial muscle, thoracic cavity)	2 × 107 transducing units	AAV6-shSOD	–	83% in vivo	No effect