title: Self-assembling nucleic acid delivery vehicles via linear, water-soluble, cyclodextrin-containing polymers.
abstract: Non-viral (synthetic) nucleic acid delivery systems have the potential to provide for the practical application of nucleic acid-based therapeutics. We have designed and prepared a tunable, non-viral nucleic acid delivery system that self-assembles with nucleic acids and centers around a new class of polymeric materials; namely, linear, water-soluble cyclodextrin-containing polymers. The relationships between polymer structure and gene delivery are illustrated, and the roles of the cyclodextrin moieties for minimizing toxicity and forming inclusion complexes in the self-assembly processes are highlighted. This vehicle is the first example of a polymer-based gene delivery system formed entirely by self-assembly.
authors.affiliation: Chemical Engineering, California Institute of Technology, Pasadena, CA 91125, USA. mdavis@cheme.caltech.edu
authors.forename: M E
authors.initials: ME
authors.lastname: Davis
authors.forename: S H
authors.initials: SH
authors.lastname: Pun
authors.forename: N C
authors.initials: NC
authors.lastname: Bellocq
authors.forename: T M
authors.initials: TM
authors.lastname: Reineke
authors.forename: S R
authors.initials: SR
authors.lastname: Popielarski
authors.forename: S
authors.initials: S
authors.lastname: Mishra
authors.forename: J D
authors.initials: JD
authors.lastname: Heidel
mesh_terms: D000818:Animals; D002482:Cellulose / Q000737:chemistry* / Q000633:toxicity; D003505:Cyclodextrins / Q000737:chemistry* / Q000633:toxicity; D004337:Drug Carriers / Q000737:chemistry* / Q000633:toxicity; D009696:Nucleic Acids / Q000008:administration & dosage* / Q000737:chemistry* / Q000235:genetics / Q000378:metabolism; D012995:Solubility; D014867:Water / Q000737:chemistry*
chemical_list: D003505:Cyclodextrins; D004337:Drug Carriers; D009696:Nucleic Acids; C041953:cyclodextrin polymer; D014867:Water; D002482:Cellulose