Premature-termination-codons readthrough compounds
Premature termination codons readthrough compounds, composition thereof, and methods of making and using the same are provided.
1. A compound having the ability to increase read through of premature termination codons (PTCs) in RNA, a pharmaceutically acceptable salt thereof, having a structure of formula (VIII):
wherein:
X is O;
R 3 is an ortho, meta, or para group and is a hydrogen, halo, or nitro; and
R 4 is hydrogen, —C(O)H, halo, or a C1-C6 group.
2. A compound having the ability to read through premature termination codons (PTCs) in RNA, a pharmaceutically acceptable salt thereof, having a structure of formula (IX):
3. The compound of claim 1 where the compound is selected from:
4. The compound of claim 1 , having one of the following formulas:
5. A composition comprising the compound of claim 1 and a pharmaceutically acceptable carrier.
6. A method of treating or ameliorating a medical condition associated with premature termination codons (PTCs) in RNA, comprising: administering to a subject a compound according to claim 1 , wherein the subject has an ATM gene that encodes one or more PTCs, or a DMD gene that encodes one or more PTCs.