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. 2020 Nov 28;10(4):258. doi: 10.3390/jpm10040258

Table 2.

Preclinical animal studies in mice models of SMA.

Reference. Promoter Codon Optimisation Dose vg/per Mouse Dose vg/kg of Body Weight Expression in CNS Mean Survival (Days) Adverse Events
[196] PGK Yes 4.5 × 1010 3 × 1013 SC: 80–140% of WT levels
Brain: low		 160 d (in 100% mice)
  • -

    Hyperactivity

  • -

    Tail necrosis

  • -

    Ear necrosis

  • -

    Bilateral cataract
[194] CBA No 5 × 1011 3.3 × 1014 SC: 42% of WT levels >250 d (n = 4, 1 death at d 97)
  • -

    Necrotic pinna
[197] CMV Yes 1 × 1011 6.7 × 1013 Lumbar SC: 66.5% MN
Thoracic SC: 45% MN
Cervical SC: 55% MN		 69 d (in 80% of mice)
  • -

    Short tail

  • -

    Ear necrosis

  • -

    Moderate eyelid inflammation

All mice were from the SMN delta 7 strain (SMN2+/+, SMNΔ7+/+, smn−/−) and received an intravenous injection in the facial vein at p1 of an AAV9 solution. The doses expressed in vg/kg were calculated using a mouse body weight at p1 of 1.5 g (the average weight of a p1 SMN-Delta7 pup [193]).