Pulmonary delivery: Biolabile DOPC–C12E4 conjugates were developed as nucleicacid carriers for in vitro and in vivo gene delivery. Their transfection efficiency and safety profile in mouse lung reflect their biodegradability and the surface active properties of the detergent released in situ. Thus, they might act as DNA carriers for gene therapy local administration through the airways.
肺部递送:生物不活泼的DOPC–C 12 E 4偶联物被开发为用于体外和体内基因递送的核酸载体。它们在小鼠肺中的转染效率和安全性反映了它们的生物降解能力和就地释放的洗涤剂的表面活性。因此,它们可以作为基因载体的DNA载体,通过气道进行局部治疗。
PHOSPHOLIPID-DETERGENT CONJUGATES AND USES THEREOF
申请人:Lebeau Luc
公开号:US20140243391A1
公开(公告)日:2014-08-28
The invention relates to novel compounds, in particular novel O-substituted phospholipids that are useful for the in vitro and in vivo delivery of drugs as well as nucleic acids into cells. The invention also relates to pharmaceutical compositions and supramolecular complexes comprising said compounds and the use of these compounds in therapeutic treatment, in particular in gene therapy.