PMID- 25022906
OWN - NLM
STAT- MEDLINE
DCOM- 20140917
LR  - 20240104
IS  - 1471-0064 (Electronic)
IS  - 1471-0056 (Linking)
VI  - 15
IP  - 8
DP  - 2014 Aug
TI  - Non-viral vectors for gene-based therapy.
PG  - 541-55
LID - 10.1038/nrg3763 [doi]
AB  - 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.
FAU - Yin, Hao
AU  - Yin H
AD  - David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute 
      of Technology (MIT), Cambridge, Massachusetts 02142, USA.
FAU - Kanasty, Rosemary L
AU  - Kanasty RL
AD  - 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.
FAU - Eltoukhy, Ahmed A
AU  - Eltoukhy AA
AD  - David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute 
      of Technology (MIT), Cambridge, Massachusetts 02142, USA.
FAU - Vegas, Arturo J
AU  - Vegas AJ
AD  - 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.
FAU - Dorkin, J Robert
AU  - Dorkin JR
AD  - 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.
FAU - Anderson, Daniel G
AU  - Anderson DG
AD  - 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.
LA  - eng
GR  - 5‑U54‑CA151884‑04/CA/NCI NIH HHS/United States
GR  - HHSN268201000045C/PHS HHS/United States
GR  - P30‑CA14051/CA/NCI NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, Non-P.H.S.
PT  - Review
DEP - 20140715
PL  - England
TA  - Nat Rev Genet
JT  - Nature reviews. Genetics
JID - 100962779
RN  - 63231-63-0 (RNA)
RN  - 9007-49-2 (DNA)
SB  - IM
MH  - Animals
MH  - DNA/genetics
MH  - Gene Transfer Techniques
MH  - *Genetic Therapy
MH  - Genetic Vectors/administration & dosage/*genetics
MH  - Humans
MH  - RNA/genetics
EDAT- 2014/07/16 06:00
MHDA- 2014/09/18 06:00
CRDT- 2014/07/16 06:00
PHST- 2014/07/16 06:00 [entrez]
PHST- 2014/07/16 06:00 [pubmed]
PHST- 2014/09/18 06:00 [medline]
AID - nrg3763 [pii]
AID - 10.1038/nrg3763 [doi]
PST - ppublish
SO  - Nat Rev Genet. 2014 Aug;15(8):541-55. doi: 10.1038/nrg3763. Epub 2014 Jul 15.