Calmodulin inhibitors for the treatment of ribosomal disorders and ribosomapathies
The present invention relates generally to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to methods for the use of calmodulin inhibitors and calcium channel blockers for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA).
1. A method of treating a subject with Diamond Blackfan Anemia (DBA), comprising administering an effective amount of a naphthalenesulfonamide compound to the subject.
2. The method of claim 1 , wherein the naphthalenesulfonamide compound is A-3, W-7, A-7, W-5, wherein A-3 has the following structure;
wherein W-7 (N-(6-Aminohexyl)-5-chloro-1-naphthalenesulfonamide hydrochloride) has the following structure:
wherein A-7 has the following structure:
wherein W-5 has the following structure:
3. The method of claim 2 , wherein a derivative of W-7 is N-(6-aminohexyl)-1-naphthalenesulfonamide hydrochloride or N-(6-aminohexyl)-5-chloro-2-naphthalenesulfonamide.