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. Author manuscript; available in PMC: 2015 Aug 1.
Published in final edited form as: Medchemcomm. 2014 May 15;5(8):1075–1091. doi: 10.1039/C4MD00163J

Table 2.

AGs investigated to treat PTC mutations in various diseases.

Disease Gene Mutations AGs Ref
CF CFTR Y122X GEN 136
G521X NB124 141
G542X AMK, GEN, TOB, NB54, NB74, NB84, NB124 138, 140–142, 154, 155
R553X GEN, NB54, NB84, NB124 141
R1162X GEN, G418, NB74, NB124 136, 141, 156
W1282X GEN, NB54, NB74, NB124 136, 140–142
RTT MECP2 Y141X GEN, G418 148
R168X GEN, G418, NB54, NB74 148–151
Q170X GEN, G418 148
E205X GEN, G418 148
R255X GEN, G418 148, 149
R270X GEN, NB54 149, 151
R294X AMK, GEN, G418, NB54 148, 149, 151, 153
SMA SMN1 SMNΔ7 TC001, TC003, TC007, TC032, JL022, JL023
Usher syndrome PCDH15 R3X GEN, G418, PAR, NB54, NB74, NB84 142, 157
R245X GEN, G418, PAR, NB54, NB74, NB84 142, 157
R643X GEN, G418, PAR 157
R929X GEN, G418, PAR 157
HS IDUA Q70X GEN, NB54, NB74, NB84 142
W402X AMK, GEN, TOB 158
Factor VII deficiency FVII K316X W364X GEN GEN 159 159
Obesity MC4R W16X G418 160
Y35X G418 160
Cancer p53 R192X GEN, G418 161
R213X GEN, G418 161
E298X GEN, G418 161
APC R213X GEN 162
L360X GEN 162
S811X GEN 162
R1114X GEN 162
Q1131X GEN 162
Q1428X GEN 162
R1450X GEN 162
DMD DMD E1593X GEN 163
Q60X
Q988X
mdx
Q1240X
Q1143X
E2726X
Q1437X
Q2125X
Q3149X
W651X
L1417X
Q2522X
E931X
Q2264X
Q673X
R1326X
C967X
R3085X
R744X
R3190X
R1549X
R1967X
R3381X
R2098X
R145X
S319X