Table 1.
Protein | Proteinopathy | Antibody format | Delivery platform | Model | Reference |
---|---|---|---|---|---|
α-synuclein* | Parkinson's disease and dementia with lewy bodies | VHH | rAAV5/ICV | Sprague–Dawley rats | Chatterjee et al. (11) |
α-synuclein* | scFv | rAAV1/ICV | DAT-Cre rats | Chen et al. (12) | |
Tau* | Frontotemporal dementia | Full-length | rAAVrh.10/ICV | Tau P301S mice | Liu et al. (13) |
Aβ* | Alzheimer's disease | scFv | rAAV1/ICV | 3xTg-AD mice | Ryan et al. (14) |
Aβ* | scFv | rAAV1/ICV | TgCRND8 mice | Levites et al. (15) | |
Aβ* | scFv | rAAV2/ICV | Tg2576 mice | Fukuchi et al. (16) | |
Aβ* | scFv | rAAV/ICV | 3xTg-AD mice | Sudol et al. (17) | |
Aβ* | scFv | rAAV5/ICV | APPswe/PS1dE9 mice | Kou et al. (18) | |
Aβ* | Full-length | rAAV1/IM | Tg2576 mice | Shimada et al. (19) | |
Aβ* | scFv | rAAV1/IM | APPswe/PS1dE9 mice | Yang et al. (20) | |
Aβ* | scFv-IgG** | rAAV1/ICV | APP/PS1 mice | Elmer et al. (21) | |
Tau* | Full-length | rAAVrh.10/ICV | Tau P301S mice | Allen et al. (22) | |
Tau* | scFv | rAAV8/ICV | Tau P301S mice | Ising et al. (23) | |
Tau* | scFv | rAAV5/ICV | JNPL3 mice | Vitale et al. (24) | |
Tau* | scFv | rAAV1/ICV | rTg4510 mice | Goodwin et al. (25) | |
TDP-43* | Amyotrophic lateral sclerosis | scFv | rAAV9/IT | TDP-43A315T mice | Pozzi et al. (26) |
SOD-1* | scFv | rAAV1/IT | SOD1G93A mice | Patel et al. (27) | |
SOD-1* | scFv | rAAV9/IV | SOD1G93A mice | Ghadge et al. (28) | |
PrPC | Prion disease | scFv | rAAV2/ICV | TSE mice | Wuertzer et al. (29) |
Huntingtin* | Huntington's disease | scFv | rAAV1/ICV | B6.HDR6/1 mice | Snyder-Keller et al. (30) |
Huntingtin* | scFv | rAAV6/ICV | R6/2_Tg mice | Amaro and Henderson (31) |
Evidence for prion-like cell to cell transmission.
Engineered for Fc effector function.
ICV, Intracerebroventricular; IM, Intramuscular; IT, Intrathecal; IV, Intravenous; TDP-43, Transactive response DNA binding protein-43; SOD1, Superoxide dismutase 1.