Table 2.

Pathways affected by compounds used for potential SMA therapy

Treatment	Process	Models used	References
Histone deacetylase inhibitors
Trichostatin A	Transcription	Δ7 SMA mice	[68,69]
Suberoylanilide hydroxamic acid	Transcription	Taiwanese mice	[80]
Sodium butyrate	Transcription	Taiwanese mice	[81]
Valproic acid	Transcription	Taiwanese mice	[90]
Translation read-through compounds
G418 (geneticin)	Translation	Δ7 SMA mice	[65]
TC007	Translation	Δ7 SMA mice	[66,67]
Quinazolines
RG3039	Transcription	Δ7 SMA mice Taiwanese mice 2B	[63] [79] [79]
D156844	Transcription	Δ7 SMA mice	[64]
Other small molecules that increase SMN2 expression
LDN-76070	Transcription	Δ7 SMA mice	[54]
Indoprofen	Splicing and/or translation	Line 89	[109]
Antibiotics
Ceftriaxone	Cell signaling	Δ7 SMA mice	[60]
PTK-SMA1 (tetracycline-like)	Splicing	Taiwanese mice	[110]
Signal transducing molecules
Sodium vanadate	Splicing	Taiwanese mice	[117]
Neuroprotective compounds
BIP-135	Cell signaling	Δ7 SMA mice	[58]
N-Methyl-D-aspartic acid	Cell signaling	Δ7 SMA mice	[59]
Riluzole	Cell signaling	F7 or exon 7 floxed	[121]
Polypeptides and proteins
Insulin-like growth factor 1	Cell signaling	Δ7 SMA mice	[57]
Follistatin	Cell signaling	Δ7 SMA mice	[61]
Prolactin	Transcription	Δ7 SMA mice	[62]
Gene therapy and trans-splicing
SMN1 gene therapy	SMN-associated functions	Δ7 SMA mice Taiwanese mice	[70–75] [82]
Trans-splicing	Splicing	Δ7 SMA mice	[133]
Stem cell based therapy of SMA
Stem cell transplantation	SMN-associated functions	Δ7 SMA mice	[55,56]
SMN-independent treatment strategies
Forced running	Multiple	Δ7 SMA mice Taiwanese mice	[148] [78]
ASO-based therapy
Antisense oligonucleotides	Splicing	Δ7 SMA mice Taiwanese mice	[50–53] [76,77]