PTC258 |
| Catalog No.GC71048 |
PTC258 est un modulateur d'épissage spécifique et actif par voie orale du gène elp1 (allonge Somatic Complex Protein 1).
Products are for research use only. Not for human use. We do not sell to patients.
Cas No.: 2476724-74-8
Sample solution is provided at 25 µL, 10mM.
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PTC258 (0.01 nM-0.01 μM; 48 h) efficiently increases full-length ELP1 mRNA and protein in Familial Dysautonomia (FD) patient fibroblasts[1].
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].
Average Rating: 5 (Based on Reviews and 30 reference(s) in Google Scholar.)
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