PMID- 26718856
OWN - NLM
STAT- MEDLINE
DCOM- 20180105
LR  - 20180228
IS  - 1873-4995 (Electronic)
IS  - 0168-3659 (Linking)
VI  - 240
DP  - 2016 Oct 28
TI  - Materials for non-viral intracellular delivery of messenger RNA therapeutics.
PG  - 227-234
LID - S0168-3659(15)30283-2 [pii]
LID - 10.1016/j.jconrel.2015.12.032 [doi]
AB  - Though therapeutics based on messenger RNA (mRNA) have broad potential in 
      applications such as protein replacement therapy, cancer immunotherapy, and 
      genomic engineering, their effective intracellular delivery remains a challenge. 
      A chemically diverse suite of delivery materials with origins as materials for 
      cellular transfection of DNA and small interfering RNAs (siRNAs) has recently 
      been reported to have promise as non-viral delivery agents for mRNA. These 
      materials include covalent conjugates, protamine complexes, nanoparticles based 
      on lipids or polymers, and hybrid formulations. This review will highlight the 
      use of delivery materials for mRNA, with a specific focus on their mechanisms of 
      action, routes of administration, and dosages. Additionally, strategies in which 
      these materials can be adapted and optimized to address challenges specific to 
      mRNA delivery are also discussed. The technologies included have shown varying 
      promise for therapeutic use, specifically having been used to deliver mRNA in 
      vivo or exhibiting characteristics that could make in vivo use a possibility. In 
      so doing, it is the intention of this review to provide a comprehensive look at 
      the progress and possibilities in applying nucleic acid delivery technology 
      specifically toward the emerging area of mRNA therapeutics.
CI  - Copyright (c) 2015 Elsevier B.V. All rights reserved.
FAU - Kauffman, Kevin J
AU  - Kauffman KJ
AD  - Department of Chemical Engineering, Massachusetts Institute of Technology, 
      Cambridge, 02139, USA; David H. Koch Institute for Integrative Cancer Research, 
      Massachusetts Institute of Technology, Cambridge, 02139, USA.
FAU - Webber, Matthew J
AU  - Webber MJ
AD  - David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute 
      of Technology, Cambridge, 02139, USA; Department of Anesthesiology, Boston 
      Children's Hospital, Boston, 02122, USA.
FAU - Anderson, Daniel G
AU  - Anderson DG
AD  - Department of Chemical Engineering, Massachusetts Institute of Technology, 
      Cambridge, 02139, USA; David H. Koch Institute for Integrative Cancer Research, 
      Massachusetts Institute of Technology, Cambridge, 02139, USA; Department of 
      Anesthesiology, Boston Children's Hospital, Boston, 02122, USA; Institute for 
      Medical Engineering and Science, Massachusetts Institute of Technology, 
      Cambridge, 02139, USA; Harvard-MIT Division of Health Science and Technology, 
      Massachusetts Institute of Technology, Cambridge, 02139, USA. Electronic address: 
      dgander@mit.edu.
LA  - eng
GR  - DP1 CA174420/CA/NCI NIH HHS/United States
PT  - Journal Article
PT  - Review
DEP - 20151221
PL  - Netherlands
TA  - J Control Release
JT  - Journal of controlled release : official journal of the Controlled Release 
      Society
JID - 8607908
RN  - 0 (Polymers)
RN  - 0 (Protamines)
RN  - 0 (RNA, Messenger)
SB  - IM
MH  - Animals
MH  - Drug Delivery Systems/*methods/trends
MH  - Genetic Therapy/*methods/trends
MH  - Humans
MH  - Intracellular Fluid/*drug effects/metabolism
MH  - Polymers/administration & dosage/metabolism
MH  - Protamines/administration & dosage/genetics/metabolism
MH  - RNA, Messenger/*administration & dosage/genetics/metabolism
OTO - NOTNLM
OT  - Drug delivery
OT  - Endosomal escape
OT  - Gene therapy
OT  - Genome editing
OT  - Immunoengineering
OT  - mRNA
EDAT- 2016/01/01 06:00
MHDA- 2018/01/06 06:00
CRDT- 2016/01/01 06:00
PHST- 2015/09/03 00:00 [received]
PHST- 2015/12/14 00:00 [revised]
PHST- 2015/12/18 00:00 [accepted]
PHST- 2016/01/01 06:00 [pubmed]
PHST- 2018/01/06 06:00 [medline]
PHST- 2016/01/01 06:00 [entrez]
AID - S0168-3659(15)30283-2 [pii]
AID - 10.1016/j.jconrel.2015.12.032 [doi]
PST - ppublish
SO  - J Control Release. 2016 Oct 28;240:227-234. doi: 10.1016/j.jconrel.2015.12.032. 
      Epub 2015 Dec 21.