This invention generally relates to a group of novel chemical compositions and their use for formulating RNA- and/or DNA-based medicine drugs/vaccines into stable compound complexes useful for both in-vitro and in-vivo delivery. Particularly, the present invention teaches the synthesis of a group of novel trimethylglycyl chemicals and their use for formulating cosmetic, therapeutic- and/or pharmaceutical-grade nucleic acid compositions, including but not limited microRNA precursors (pre-miRNA/miRNA), small hairpin RNAs (shRNA), short interfering RNAs (siRNA), ribozymes, antisense oligonucleotides, RNA-DNA hybrids and DNA-based vectors/vaccines, with or without modification, into delivery complexes, which can then be absorbed by cells in vivo, ex vivo and/or in vitro through an active mechanism of endocytosis via acetylcoline receptors for releasing the therapeutic and pharmaceutical effects of the formulated nucleic acid compositions.
本发明一般涉及一组新型
化学组合物,及其用于将基于 RNA 和/或 DNA 的药物/疫苗配制成稳定的化合物复合物,既可用于体外给药,也可用于体内给药。特别是,本发明教导了一组新型三甲基甘
氨酰
化学品的合成及其用于配制化妆品级、治疗级和/或药物级核酸组合物的方法,包括但不限于 microRNA 前体(pre-miRNA/miRNA)、小发夹 RNA(shRNA)、短干扰 RNA(siRNA)、核酶、它们可以通过
乙酰胆碱受体的内吞活性机制被体内、体外和/或体外细胞吸收,从而释放所配制核酸组合物的治疗和药效。