Literature DB >> 30846391

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery.

Piotr S Kowalski1, Arnab Rudra2, Lei Miao1, Daniel G Anderson3.   

Abstract

mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellular delivery methods. However, broad application of mRNA is still limited by the need for improved delivery systems. In this review, we discuss the challenges for clinical translation of mRNA-based therapeutics, with an emphasis on recent advances in biomaterials and delivery strategies, and we present an overview of the applications of mRNA-based delivery for protein therapy, gene editing, and vaccination.
Copyright © 2019. Published by Elsevier Inc.

Entities:  

Keywords:  CRISPR; biomaterials; clinical trial; gene editing; gene therapy; lipid nanoparticles; mRNA delivery; mRNA nanoparticles; mRNA vaccine; protein replacement

Mesh：

Substances：

Year:  2019        PMID： 30846391      PMCID： PMC6453548          DOI： 10.1016/j.ymthe.2019.02.012

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


  185 in total

1.  In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency.

Authors:  Azita J Mahiny; Alexander Dewerth; Lauren E Mays; Mohammed Alkhaled; Benedikt Mothes; Emad Malaeksefat; Brigitta Loretz; Jennifer Rottenberger; Darina M Brosch; Philipp Reautschnig; Pacharapan Surapolchai; Franziska Zeyer; Andrea Schams; Melanie Carevic; Martina Bakele; Matthias Griese; Matthias Schwab; Bernd Nürnberg; Sandra Beer-Hammer; Rupert Handgretinger; Dominik Hartl; Claus-Michael Lehr; Michael S D Kormann
Journal:  Nat Biotechnol       Date:  2015-05-18       Impact factor: 54.908

2.  Cationic lipids enhance siRNA-mediated interferon response in mice.

Authors:  Zheng Ma; Jiang Li; Fengtian He; Annette Wilson; Bruce Pitt; Song Li
Journal:  Biochem Biophys Res Commun       Date:  2005-05-13       Impact factor: 3.575

3.  Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.

Authors:  Akin Akinc; William Querbes; Soma De; June Qin; Maria Frank-Kamenetsky; K Narayanannair Jayaprakash; Muthusamy Jayaraman; Kallanthottathil G Rajeev; William L Cantley; J Robert Dorkin; James S Butler; Liuliang Qin; Timothy Racie; Andrew Sprague; Eugenio Fava; Anja Zeigerer; Michael J Hope; Marino Zerial; Dinah W Y Sah; Kevin Fitzgerald; Mark A Tracy; Muthiah Manoharan; Victor Koteliansky; Antonin de Fougerolles; Martin A Maier
Journal:  Mol Ther       Date:  2010-05-11       Impact factor: 11.454

4.  Systemic delivery of factor IX messenger RNA for protein replacement therapy.

Authors:  Suvasini Ramaswamy; Nina Tonnu; Kiyoshi Tachikawa; Pattraranee Limphong; Jerel B Vega; Priya P Karmali; Pad Chivukula; Inder M Verma
Journal:  Proc Natl Acad Sci U S A       Date:  2017-02-15       Impact factor: 11.205

5.  An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo.

Authors:  Bin Li; Xiao Luo; Binbin Deng; Junfeng Wang; David W McComb; Yimin Shi; Karin M L Gaensler; Xu Tan; Amy L Dunn; Bryce A Kerlin; Yizhou Dong
Journal:  Nano Lett       Date:  2015-11-06       Impact factor: 11.189

Review 6.  Delivery approaches for CRISPR/Cas9 therapeutics in vivo: advances and challenges.

Authors:  D C Luther; Y W Lee; H Nagaraj; F Scaletti; V M Rotello
Journal:  Expert Opin Drug Deliv       Date:  2018-09-12       Impact factor: 6.648

7.  Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system.

Authors:  F DeRosa; B Guild; S Karve; L Smith; K Love; J R Dorkin; K J Kauffman; J Zhang; B Yahalom; D G Anderson; M W Heartlein
Journal:  Gene Ther       Date:  2016-06-30       Impact factor: 5.250

8.  CRISPR RNA-guided activation of endogenous human genes.

Authors:  Morgan L Maeder; Samantha J Linder; Vincent M Cascio; Yanfang Fu; Quan H Ho; J Keith Joung
Journal:  Nat Methods       Date:  2013-07-25       Impact factor: 28.547

Review 9.  A novel, disruptive vaccination technology: self-adjuvanted RNActive(®) vaccines.

Authors:  Karl-Josef Kallen; Regina Heidenreich; Margit Schnee; Benjamin Petsch; Thomas Schlake; Andreas Thess; Patrick Baumhof; Birgit Scheel; Sven D Koch; Mariola Fotin-Mleczek
Journal:  Hum Vaccin Immunother       Date:  2013-06-04       Impact factor: 3.452

10.  Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery.

Authors:  Jianbin Wang; Joshua J DeClercq; Samuel B Hayward; Patrick Wai-Lun Li; David A Shivak; Philip D Gregory; Gary Lee; Michael C Holmes
Journal:  Nucleic Acids Res       Date:  2015-11-02       Impact factor: 16.971
View more
  203 in total

1.  In vitro-Transcribed mRNA Therapeutics: Out of the Shadows and Into the Spotlight.

Authors:  Katalin Karikó
Journal:  Mol Ther       Date:  2019-03-21       Impact factor: 11.454

2.  mTORC1/rpS6 signaling complex modifies BTB transport function: an in vivo study using the adjudin model.

Authors:  Ming Yan; Linxi Li; Baiping Mao; Huitao Li; Stephen Y T Li; Dolores Mruk; Bruno Silvestrini; Qingquan Lian; Renshan Ge; C Yan Cheng
Journal:  Am J Physiol Endocrinol Metab       Date:  2019-05-21       Impact factor: 4.310

3.  siRNA Design and GalNAc-Empowered Hepatic Targeted Delivery.

Authors:  Mei Lu; Mengjie Zhang; Bo Hu; Yuanyu Huang
Journal:  Methods Mol Biol       Date:  2021

Review 4.  The promise of mRNA vaccines: a biotech and industrial perspective.

Authors:  Nicholas A C Jackson; Kent E Kester; Danilo Casimiro; Sanjay Gurunathan; Frank DeRosa
Journal:  NPJ Vaccines       Date:  2020-02-04       Impact factor: 7.344

5.  Formulation and Delivery Technologies for mRNA Vaccines.

Authors:  Chunxi Zeng; Chengxiang Zhang; Patrick G Walker; Yizhou Dong
Journal:  Curr Top Microbiol Immunol       Date:  2020-06-02       Impact factor: 4.291

6.  mRNA Delivery for Therapeutic Anti-HER2 Antibody Expression In Vivo.

Authors:  Yulia Rybakova; Piotr S Kowalski; Yuxuan Huang; John T Gonzalez; Michael W Heartlein; Frank DeRosa; Derfogail Delcassian; Daniel G Anderson
Journal:  Mol Ther       Date:  2019-05-18       Impact factor: 11.454

7.  bioPROTACs as versatile modulators of intracellular therapeutic targets including proliferating cell nuclear antigen (PCNA).

Authors:  Shuhui Lim; Regina Khoo; Khong Ming Peh; Jinkai Teo; Shih Chieh Chang; Simon Ng; Greg L Beilhartz; Roman A Melnyk; Charles W Johannes; Christopher J Brown; David P Lane; Brian Henry; Anthony W Partridge
Journal:  Proc Natl Acad Sci U S A       Date:  2020-03-02       Impact factor: 11.205

8.  RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges.

Authors:  Ai-Ming Yu; Young Hee Choi; Mei-Juan Tu
Journal:  Pharmacol Rev       Date:  2020-10       Impact factor: 25.468

9.  Delivery of hepatocyte growth factor mRNA from nanofibrillar scaffolds in a pig model of peripheral arterial disease.

Authors:  Tatiana S Zaitseva; Guang Yang; Dimitris Dionyssiou; Maedeh Zamani; Steve Sawamura; Eduard Yakubov; James Ferguson; Richard L Hallett; Dominik Fleischmann; Michael V Paukshto; Ngan F Huang
Journal:  Regen Med       Date:  2020-08-10       Impact factor: 3.806

10.  Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening.

Authors:  Pedro P G Guimaraes; Rui Zhang; Roman Spektor; Mingchee Tan; Amanda Chung; Margaret M Billingsley; Rakan El-Mayta; Rachel S Riley; Lili Wang; James M Wilson; Michael J Mitchell
Journal:  J Control Release       Date:  2019-10-31       Impact factor: 9.776
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.