Tìm theo
PTC124
Các tên gọi khác (1) :
  • EC-000.2051
Thuốc Gốc
Small Molecule
CTHH: C15H9FN2O3
PTK: 284.242
PTC124 is a novel, orally administered drug that targets nonsense mutations and is being investigated initially as a treatment for Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF), with the potential to treat a number of other genetic disorders caused by nonsense mutations.
Nhận Dạng Quốc Tế & Đặc Tính Hóa Học
Công thức hóa học
C15H9FN2O3
Phân tử khối
284.242
Monoisotopic mass
284.059720369
InChI
InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)
InChI Key
InChIKey=OOUGLTULBSNHNF-UHFFFAOYSA-N
IUPAC Name
3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
Traditional IUPAC Name
ataluren
SMILES
OC(=O)C1=CC=CC(=C1)C1=NOC(=N1)C1=CC=CC=C1F
Độ hòa tan
1.17e-01 g/l
logP
3.92
logS
-3.4
pKa (strongest acidic)
3.9
pKa (Strongest Basic)
-1.6
PSA
76.22 Å2
Refractivity
94.66 m3·mol-1
Polarizability
27.66 Å3
Rotatable Bond Count
3
H Bond Acceptor Count
4
H Bond Donor Count
1
Physiological Charge
-1
Number of Rings
3
Bioavailability
1
Rule of Five
true
Ghose Filter
true
Cơ Chế Tác Dụng : PTC124 is a novel, orally administered drug that targets nonsense mutations and is being investigated initially as a treatment for Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF), with the potential to treat a number of other genetic disorders caused by nonsense mutations. PTC124 allowed the cellular machinery to bypass the nonsense mutation, continue the translation process, and thereby restore the production of a full-length, functional protein. The research on the effects of PTC124 on the translation and stability of nonsense-containing mRNA in vitor show that PTC124 promoted readthrough at each of the nonsense codons, showing maximal activity with UGA, while having no effect on mRNA levels. Unlike the stable cell line assays, PTC124 did not discriminate significantly between the UAG and UAA mRNAs. PTC124 was a more potent nonsense-suppressing agent than gentamicin, and exhibited 4- to 15-fold stimulation of in vitro readthrough relative to the controls at levels similar to those in the stable cell reporter assays. These results indicate that PTC124 modulates termination efficiency at premature nonsense codons.
Dược Động Học :

▧ Half Life :
3-6 hours
Chỉ Định : Investigated for use/treatment in cystic fibrosis and muscular dystrophy.
Tài Liệu Tham Khảo Thêm
drugbank
http://www.drugbank.ca/drugs/DB05016
PubChem Compound
http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?sid=11219835
PubChem Substance
http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?sid=29203630
ChemSpider
http://www.chemspider.com/Chemical-Structure.9394889.html
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