GUILD, BRAYDON CHARLES,DEROSA, FRANK,HEARTLEIN, MICHAEL
申请号：
CL2013003478
公开号：
CL2013003478A1
申请日:
2013.12.04
申请国别(地区):
CL
年份:
2014
代理人:
摘要：
The component includes (a) at least one RNM molecule, at least one of which encodes a peptide of a functional Secretary (preferably eritropoyetin or functional Galaxy)；(b) Transfer vehicles, including a liquid nanomolecule (less than 100 nm), consisting of one or more cationic lipids, one or more non cationic lipids, one or more cholesterol based lipids, and one or more mucilage modified lipids. More preferably, RNM molecules include a change that improves their stability (preferably including cap1 structure or a-end). In one form, it is composed of gonorrhea (recombinant or not).It can also include a preparation that can transfer RNM molecules to target cells.The components used include (a) at least one RNM for encrypting functional Secretary peptides (preferably those listed in R18, with cap added in end 5 and a polymerized Thrace added in end 3), and (b) a transfer vehicle containing lipid particles, 1. To prepare effective drugs for the treatment of functional peptide deficiency；A method of producing a functional Secretary polypeptide in a target cell, including managing the claimed components, after which RNM becomes a target cell, thereby producing a functional polypeptide.Composición que comprende a) al menos una molécula de ARNm donde al menos parte de la misma codifica un polipéptido secretado funcional (preferentemente eritropoyetina o a-galactosidasa funcional)； y b) un vehículo de transferencia que comprende una nanopartócula lipídica (de un tamaño menor a 100 nm) que comprende uno o más lípidos catiónicos, uno o más lípidos no catiónicos, uno o más lípidos basados en colesterol, y uno o más lípidos modificados por PEG. Preferentemente la molécula de ARNm comprende una modificación que aumenta su estabilidad (de preferencia la inclusión de una estructura Cap1 o de un extremo poli A). En una modalidad la composición se encuentra en forma liofilizada (reconstituida o no), y puede comprender un agente que facilita la transferencia de la molécula de ARN
