Biological Activity
PTC-258 2HCl 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.
In Vitro
No in vitro data are provided in the current product source. Contact us for further information.
In Vivo
No in vivo data are provided in the current product source. Contact us for further information.
References & source
[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.
Published experimental findings depend on the model and assay conditions. They do not guarantee equivalent performance in every application. For laboratory research use only.