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Chemical Structure : PTC258
Catalog No.: PC-49817Not For Human Use, Lab Use Only.
PTC-258 (PTC258) is a highly potent, specific and orally active ELP1 splicing modulator, efficiently increases full-length ELP1 mRNA and protein in FD patient fibroblasts (EC2X ELP1 protein=0.34 nM).
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PTC-258 (PTC258) is a highly potent, specific and orally active ELP1 splicing modulator, efficiently increases full-length ELP1 mRNA and protein in FD patient fibroblasts (EC2X ELP1 protein=0.34 nM).
PTC-258 is about 30,000 times more potent than kinetin (EC2X ELP1 protein=10,000 nM) and about 1,000 times more potent than BPN15477 (EC2X ELP1 protein=340 nM).
PTC258 (3-24 mg/kg/day, oral) increased full-length ELP1 transcript in a dose dependent manner, led to at least a 5-fold increase in functional ELP1 protein in the brain, trigeminal, liver, and quadrice, in TgFD9 transgenic mouse.
Oral administration of PTC258 starting at birth prevented gait ataxia and retinal degeneration in the phenotypic FD mouse, prevented neuronal loss in FD DRG and retina, increased the amount of ELP1 in CNS and PNS.
Oral administration of PTC258 significantly improved the thickness of RNFL and GCIPL by promoting RGC survival.
| M.Wt | 351.91 | |
| Formula | C16H18ClN3S2 | |
| Appearance | Solid | |
| Storage |
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| Solubility |
10 mM in DMSO |
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1. Elisabetta Morini, et al. Am J Hum Genet. 2023 Feb 15;S0002-9297(23)00044-7.
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