System for delivering therapeutic agents into living cells and cells nuclei
The present invention relates to a novel delivery system for delivering therapeutic agents into living cells, and more particularly, to novel chemical moieties that are designed capable of targeting and/or penetrating cells or other targets of interest and further capable of binding therapeutic agents to be delivered to these cells, and to delivery systems containing same.
1. A compound represented by the structure of formula XXX:
wherein
R 1 and R 2 are each independently selected from a C 1 -C 6 linear or branched alkyl, C 3 -C 7 cycloalkyl, or R 1 and R 2 are joined together to form a 4-7 membered heterocyclic ring;
R 3 is a C 1 -C 6 linear or branched alkyl, CH 3 , CH 2 CH 2 CN, CH 2 CH 2 SO 2 Ar or CH 2 CH 2 SO 2 CH 3 ;
R 4 is an alcohol protecting group, wherein said alcohol protecting group is: trityl (triphenylmethyl, Tr), Methoxytrityl [(4-methoxyphenyl)diphenylmethyl, MMT), Dimethoxytrityl [bis-(4-methoxyphenyl)phenylmethyl] (DMT), Acetyl (Ac), Benzoyl (Bz), Benzyl (Bn, Bul), β-Methoxyethoxymethyl ether (MEM), Methoxymethyl ether (MOM), p-Methoxybenzyl ether (PMB), Methylthiomethyl ether, Pivaloyl (Piv), Tetrahydropyranyl (THP), Silyl ether, methyl ether or ethoxyethyl ether;
R 5 is an amine protecting group, wherein said amine protecting group is: TFA (trifluoroacetamide), FMOC (9-Fluorenylmethyloxycarbonyl), FMS [(9H-Fluoren-9-yl)methanesulfonyl], acetyl (Ac) or benzoyl (Bz).
2. The compound of claim 1 , wherein R 1 and R 2 are C 1 -C 6 linear or branched alkyl.
3. The compound of claim 1 , wherein R 3 is CH 2 CH 2 CN.
4. The compound of claim 1 , wherein R 4 is Dimethoxytrityl [bis-(4-methoxyphenyl)phenylmethyl)] (DMT).
5. The compound of claim 1 , wherein R 5 is trifluoroacetamide (TFA).
6. The compound of claim 1 , wherein said compound is represented by the following structure: