| 摘要 |
<p>This invention herein describes pharmaceutical compositions and methods for targeted delivery of functional genes into cells and tissues in vivo. The invention discloses DNA:lipid complexes, methods of making such complexes and methods of using such complexes for facilitating the targeted delivery and entry of recombinant expression constructs into cells and tissues in vivo, and particularly delivery of such recombinant expression constructs to lung cells and tissues. The delivery vehicle for the targeting of recombinant construct encoding the gene of interest is composed of a mixture of a cationic lipid of formula (A): wherein Z is alkyl or alkoxyalkyl, n is an integer from 1 to 4 inclusive, R or R1 are C11-29 straight chain aliphatic hydrocarbyl groups and R2 is hydrogen or lower alkyl, together with neutral lipids (e.g. DOPE, cholesterol). The gene to be targeted is preferably CFTR gene for cystic fibrosis and the targeted tissue is lung.</p> |
