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. 2021 Jun 21;2(4):1158–1166. doi: 10.1039/d1cb00110h

Summary of candidate therapeutics directly targeting TDP-43.

Name Therapeutic type Targeted site Cell Fly Mice
3B12A Antibody Misfolded RRM2 Clearance of pathological TDP-43 in HEK293A cells N/A Clearance of TDP-43 aggregates in newborn mice transduced with TDP-43mNLS,C173S/C175S
VH7Vk9 Antibody Misfolded RRM1 Induced proteasome and autophagy-driven degradation of TDP-43 in HEK293 cells N/A Improved cognitive function in TDP-43G348C mice
Liu “A, B, C, D, E” Peptide CTD Reduced aggregation of TDP-43 in HeLa cells but not toxicity N/A N/A
Gao “D4” Peptide CTD Reduced TDP-43 levels in an N2a cell line overexpressing TDP-43 Recapitulated in Drosophila overexpressing TDP-43 N/A
AIM4 Small molecule CTD Reduced TDP-43 inclusions in a yeast S. cerevisiae model N/A N/A
rTRD01 Small molecule RRM1/2 N/A ALS Drosophila model results in reduced locomotor defects N/A
nTRD22 Small molecule NTD Reduced TDP-43 protein levels in rodent primary motor neurons Mitigated motor impairment in Drosophila model of ALS N/A