The present invention provides, in part cystine cationic lipid compounds of formula A, and sub-formulas thereof: or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.
本发明部分提供了公式A的半胱
氨酸阳离子脂质化合物及其子公式:或其药用可接受盐。本文提供的化合物可用于传递和表达mRNA和编码蛋白质,例如作为脂质体传递载体的组成部分,并因此可用于治疗各种疾病、紊乱和病况,如与一个或多个蛋白质缺乏相关的疾病。