title: Non-viral vectors for gene-based therapy.
abstract: Gene-based therapy is the intentional modulation of gene expression in specific cells to treat pathological conditions. This modulation is accomplished by introducing exogenous nucleic acids such as DNA, mRNA, small interfering RNA (siRNA), microRNA (miRNA) or antisense oligonucleotides. Given the large size and the negative charge of these macromolecules, their delivery is typically mediated by carriers or vectors. In this Review, we introduce the biological barriers to gene delivery in vivo and discuss recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems, some of which are currently undergoing testing in clinical trials. The diversity of these systems highlights the recent progress of gene-based therapy using non-viral approaches.
authors.affiliation: David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA.
authors.forename: Hao
authors.initials: H
authors.lastname: Yin
authors.affiliation: 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA. [2] Department of Chemical Engineering, MIT, Cambridge, Massachusetts 02142, USA.
authors.forename: Rosemary L
authors.initials: RL
authors.lastname: Kanasty
authors.affiliation: David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA.
authors.forename: Ahmed A
authors.initials: AA
authors.lastname: Eltoukhy
authors.affiliation: 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA. [2] Department of Anesthesiology, Children's Hospital Boston, Boston, Massachusetts 02115, USA.
authors.forename: Arturo J
authors.initials: AJ
authors.lastname: Vegas
authors.affiliation: 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA. [2] Department of Biology, MIT, Cambridge, Massachusetts 02142, USA.
authors.forename: J Robert
authors.initials: JR
authors.lastname: Dorkin
authors.affiliation: 1] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT), Cambridge, Massachusetts 02142, USA. [2] Department of Chemical Engineering, MIT, Cambridge, Massachusetts 02142, USA. [3] Department of Anesthesiology, Children's Hospital Boston, Boston, Massachusetts 02115, USA. [4] Harvard-MIT Division of Health Sciences & Technology, MIT, Cambridge, Massachusetts 02139, USA. [5] Institute for Medical Engineering and Science, MIT, Cambridge, Massachusetts 02142, USA.
authors.forename: Daniel G
authors.initials: DG
authors.lastname: Anderson
mesh_terms: D000818:Animals; D004247:DNA / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics*; D006801:Humans; D012313:RNA / Q000235:genetics
chemical_list: D012313:RNA; D004247:DNA