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. Author manuscript; available in PMC: 2025 Mar 10.
Published in final edited form as: Adv Drug Deliv Rev. 2024 Jun 19;211:115363. doi: 10.1016/j.addr.2024.115363

Table 2.

Summary of AAV delivery by FUS-BBBO in mice, rats, and NHPs.

Animal AAV parameters FUS parameters Ref
Animal Target Serotype Gene Dose (vg/g) Microbubble (MB) Dose Frequency
(MHZ)
Pressure
(MPa)
PRF
(Hz)
Pulse length
(ms)
Duration (s)
Mouse CT, STR, HP, TH AAV9 GFP 5×108
2.5×109
1.25×1010
40–80 μL/kg 0.558
1.18
0.3
0.53–0.6
1 10 120 [130]
HP, CT AAV9 mcherry-ChR2 4×1011 vg/animal 5 μL 1.5 0.74 5 10 120 [145]
HP, SN, OB, DMV AAV9 1.25×1010 0.02 mL/kg 1.68 1.0 ± 0.2
1.1 ± 0.2
1 10 120 [146]
STR, HP, midbrain AAV9
6.7×109
1.3×109
1.5×106 MB 1.5 0.33 1 10 120 [147]
Whole brain AAV9 GFP 1.1×1011
vg/animal
8 μL/mL at 100 μL volume 1.5 0.57 10 99 150 [148]
HP AAV9 GFP 6.5×1010
1.0×1010
1.3×1011
vg/animal
3.6×108 MBs/mL 1.5 1.0 0.33 1.0
3.33
6.67
120 [149]
STR, TH AAV9 GFP 1×1011
1.3×1010
0.02 mL/kg 1.78 0.06
0.12
0.25
0.38
0.5 10 120 [150]
Large brain regions AAV9 GFP 1×1010 1.5×106 MBs 1.53 0.27 1 10 30 [151]
HP AAV9-hM4Di mcherry 1×1010 1.5×106 MBs 1.5 0.42 1 10 120 [152]
CT AAV9,
PHP.B
1×1011
vg/animal
1.5×107 MBs in 50 μL volume 1.5 0.51 5 1.0 660 [153]
CT, STR, TH AAV9,
PHP.B
GFP 1×1010
1×1011
0.02 mL/kg 1.68 to 1.78 ~0.5 – 2.0 1 10 120 [139]
HP AAV9
AAV1
mcherry-ChR2 2.48×1012
vg/animal
~2.5×107 MBs 1.5 0.45 5 6.7 60 [154]
STR, TH AAV9
AAV2-HBKO
GFP 1.7×109
3.3×109
1.7×1010
0.02 mL/kg 0.58 NA 1 10 120 [155]
STR, HP, TH, CT AAV6
AAV1/2
AAV9
GFP 3×109 0.02 mL/kg 1.68 NA 1 10 120 [156]
CT, STR, TH, HP AAV1, 2, 5, 8, 9, rg TdTomatoGFP 3.33×109
1.67×1010
0.02 mL/kg 0.58 <0.9 1 10 180 [131]
STR AAV2 hrGFP 1×109 0.3 mL/kg 1.5 0.44
0.53
0.7
1 10 120 [136]
CPu AAV1 GFP 1.1×1011
vg/animal
~2.5×107 MBs 1.5 0.45 5 20 300 [138]
CT, HP AAV1/2 GFP 3×109 0.02 mL/kg 1.68 NA 1 10 120 [157]
HP AAV9-Cu64 PKM2 2×1011
5×1011
vg/animal
2.5×108 MBs/kg 1.5 0.42
0.6
0.74
NA NA 120 [158]
Rat Left brain AAV2/1 LacZ 1.06×1011 vg/animal 0.2×109 MBs/mL 0.5 1.1 1 10 40+ [159]
STR AAV1/2 GFP 1×109 0.4×108 − 0.64×108 MBs/kg 0.97 1 10 200 [137]
STR, SN AAV2 NA 2.96×1012 − 3.94×1012
vg/animal
NA 0.58 0.4 1 10 120 [160]
NHP CT, TH, ST midbrain AAV9-PHP.eB
AAV9.2-PHP.eB
GFP 5×1013
vg/animal
4 μL/kg 0.220 1 Watts NA NA 60 to 150 [143]
CT AAV2
AAV9
EGFP
mCherry
NA 200 μL/kg 1.46 1.17 1 10 60 [144]

CT: Cortex; STR: Striatum; HP: Hippocampus; TH: Thalamus; CPu: Caudate putamen; Cd: Caudate nucleus; SN: Substantia nigra; OB: Olfactory bulb; DMV: Dorsal motor nucleus of the vagus; STN: Subthalamic nucleus; NHP: Non-human primates.