PTC258 |
Catalog No.GC71048 |
PTC258 est un modulateur d'épissage spécifique et actif par voie orale du gène elp1 (allonge Somatic Complex Protein 1).
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Cas No.: 2476724-74-8
Sample solution is provided at 25 µL, 10mM.
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].
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