Characterization of plasmids and formulations for non-viral gene therapy.
Analytical methods are described that provide a platform for systematically evaluating the effect of formulation variables in the development of DNA-lipid non-viral gene therapy complexes. The methods encompass those used to characterize the components, i.e., plasmid DNA and liposomes, as well as those applied to the complexes themselves. The focus is on physicochemical methods so that their parameters can be assessed in relationship to the functionality of the DNA-lipid complexes in vivo.
Hutchins
B
B
Process Sciences Department, Canji Inc, 3525 John Hopkins Court, San Diego, CA 92121, USA. beth.hutchins@canji.com
D000818:Animals; D002626:Chemistry, Pharmaceutical; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008081:Liposomes / Q000008:administration & dosage / Q000737:chemistry; D010957:Plasmids / Q000008:administration & dosage / Q000737:chemistry / Q000235:genetics*
D008081:Liposomes