CGT Wiki

Applicable species: mice, rats, NHP (cynomolgus monkeys) [12-56,12-57]. Dose range: mouse 2.1×10^8 vg (scAAV1, 2 μL divided into 2 branches); rat 2×10^11 GC/nerve; NHP single nerve 2.5×10^12-2.6×10^13 vg/nerve (3×10^12-2.1×10^13 vg/kg based on body weight) [12-56,12-57]. Injection volume: 2 μL for mice; 20 μL for rats; 300 μL bolus for NHP each time[12-56,12-57]. Needle: mouse glass microelectrode/Hamilton; NHP ultrasound-guided nerve block blunt needle + nerve stimulator confirmation (≥0.3 mA indicates that it is safer to be farther away from the fiber) [12-56,12-57]. Speed: NHP 100 μL/min, the distance between adjacent injection points is 3 cm (300 μL spreads about 3 cm) [12-56,12-57]. Operating points: NHP ultrasound locates nerves, and each animal is injected with 10-20 nerves; it can contain 0.1% Fast Green for visualization [12-56,12-57]. Coverage: AAV2/9 can penetrate the perineurium and transduce up to 90% of myelinated Schwann cells, evenly distributed along the 15 cm nerve length; scAAV1 is most efficiently transported retrogradely to DRG and spinal motor neurons; there are off-targets in the liver (0.9 vg/dg) and spleen (3.6 vg/dg) [12-56,12-57]. Safety: Excessive intraneural injection pressure can cause capillary collapse/ischemia/axonal damage. NHP recommends the perineural route as the first choice for clinical translation [12-56,12-57].