| Bioactivity | PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model[1]. |
| Invitro | PTC258 (0.01 nM-0.01 μM; 48 h) efficiently increases full-length ELP1 mRNA and protein in Familial Dysautonomia (FD) patient fibroblasts[1]. |
| In Vivo | PTC258 (3-24 mg/kg; p.o.; once daily for 3 months) is well tolerated. It corrects the splicing of the ELP1 transcript and significantly increases the amount of functional protein in vivo in all tissues tested, including the brain in mouse model[1]. Animal Model: |
| Name | PTC258 |
| CAS | 2476724-74-8 |
| Formula | C16H18ClN3S2 |
| Molar Mass | 351.92 |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
| Reference | [1]. Morini E, et al. Development of a novel oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia[J]. bioRxiv, 2022: 2022.11. 04.515198. |
PTC258
CAS: 2476724-74-8 F: C16H18ClN3S2 W: 351.92
PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases t
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