| abstract |
A composition for delivery of Factor VIII is described. The composition is comprised of an expression vector encoding for Factor VIII and a lipid represented by the formula: n n n where each of R 1 and R 2 is an alkyl or alkenyl chain having between 8-24 carbon atoms; n=0-20, preferably 1-20; L is selected from the group consisting of (i) —X—(C═O)—Y—, (ii) —X—(C═O)—, and (iii) —X—, where X and Y are independently selected from oxygen, NH and a direct bond; and Z is a weakly basic moiety that has a pK of less than 7.4 and greater than about 4.0. |