Bioactivity | Nesolicaftor (PTI-428) is a specific cystic fibrosis transmembrane conductance regulator (CFTR) amplifier[1]. | ||||||||||||
Target | CFTR | ||||||||||||
Invitro | Nesolicaftor (PTI-428) (30 µM, 24 h) can increase CFTR function, such as causing a larger change in inh-172 in 16HBE14o-cells expressing G542X-CFTR, also can increase in F508del-CFTR function of CFBE41o-cells expressing F508del-CFTR in a dose-dependent manner[2]. | ||||||||||||
Name | Nesolicaftor | ||||||||||||
CAS | 1953130-87-4 | ||||||||||||
Formula | C18H18N4O4 | ||||||||||||
Molar Mass | 354.36 | ||||||||||||
Appearance | Solid | ||||||||||||
Transport | Room temperature in continental US; may vary elsewhere. | ||||||||||||
Storage |
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Reference | [1]. Mijnders M, et al. Correcting CFTR folding defects by small-molecule correctors to cure cystic fibrosis. Curr Opin Pharmacol. 2017 Jun;34:83-90. [2]. Arianna Venturini, et al. Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene. Int J Mol Sci. 2021 Nov 4;22(21):11972. |